2022

Harrasser, M., Gohil, S.H., Lau, H., Della Peruta, M., Muczynski, V., Patel, D., ...Granger, D. (2022). Inducible localized delivery of an anti-PD-1 scFv enhances anti-tumor activity of ROR1 CAR-T cells in TNBC. Breast Cancer Research, 24 (1), doi:10.1186/s13058-022-01531-1
Nathwani, A.C., McIntosh, J., Sheridan, R. (2022). Liver Gene Therapy.. Hum Gene Ther, doi:10.1089/hum.2022.169
Chowdary, P., Shapiro, S., Makris, M., Evans, G., Boyce, S., Talks, K., ...Riddell, A. (2022). Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B. The New England Journal of Medicine, 387 (3), 237-247. doi:10.1056/NEJMoa2119913
Chowdary, P., Nathwani, A. (2022). Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B: The authors reply. New England Journal of Medicine, 387 (14), 1336-1337. doi:10.1056/NEJMc2210918
Brimble, M.A., Cheng, P.H., Winston, S.M., Reeves, I.L., Souquette, A., Spence, Y., ...Valentine, M. (2022). Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps. Molecular Therapy - Methods and Clinical Development, 24 280-291. doi:10.1016/j.omtm.2022.01.008

2021

Allsup, D., Howard, D., Emmerson, J., Hockaday, A., Rawstron, A., Oughton, J.B., ...Paneesha, S. (2021). COSMIC, chemotherapy plus ofatumumab at standard or mega-dose in chronic lymphocytic leukaemia, a phase II randomised study. British Journal of Haematology, doi:10.1111/bjh.17526
Edwards, K., Zaitseva, K., Sayed, U., Volpi, E.V., Nathwani, A., Gribben, J.G., ...Porakishvili, N. (2021). Expression patterns of CD180 in the lymph nodes of patients with chronic lymphocytic leukaemia. British Journal of Haematology, doi:10.1111/bjh.17680
Bolous, N.S., Chen, Y., Wang, H., Davidoff, A.M., Devidas, M., Jacobs, T.W., ...Piras, B.A. (2021). The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective. Blood, 138 (18), 1677-1690. doi:10.1182/blood.2021010864

2020

Ferrière, S., Peyron, I., Christophe, O.D., Kawecki, C., Casari, C., Muczynski, V., ...Denis, C.V. (2020). A hemophilia A mouse model for the in vivo assessment of emicizumab function. Blood, 136 (6), 740-748. doi:10.1182/blood.2019004334
Nathwani, A.C., Tuddenham, E.G.D. (2020). Haemophilia, the journey in search of a cure. 1960–2020. British Journal of Haematology, 191 (4), 573-578. doi:10.1111/bjh.17155
Sia, K.C., Fu, Z.Y., Calne, R.Y., Nathwani, A.C., Lee, K.O., Gan, S.U. (2020). Modification of a Constitutive to Glucose-Responsive Liver-Specific Promoter Resulted in Increased Efficacy of Adeno-Associated Virus Serotype 8-Insulin Gene Therapy of Diabetic Mice. Cells, 9 (11), doi:10.3390/cells9112474

2019

Nathwani, A.C. (2019). Gene therapy for hemophilia.
Gohil, S.H., Evans, R., Harasser, M., El-Kholy, M., Paredes-Moscosso, S.R., Della Peruta, M., Nathwani, A.C. (2019). Ibrutinib enhances the efficacy of ROR1 bispecific T cell engager mediated cytotoxicity in chronic lymphocytic leukaemia. British Journal of Haematology, doi:10.1111/bjh.15911
Gohil, S.H., Evans, R., Harrasser, M., El-Kholy, M., Paredes-Moscosso, S.R., Della Peruta, M., Nathwani, A.C. (2019). Ibrutinib enhances the efficacy of ROR1 bispecific T cell engager mediated cytotoxicity in chronic lymphocytic leukaemia (vol 186, pg 380, 2019). BRITISH JOURNAL OF HAEMATOLOGY, 186 (6), 908. doi:10.1111/bjh.16162
Recino, A., Gan, S.U., Sia, K.C., Sawyer, Y., Trendell, J., Kay, R., ...Notaridou, M. (2019). Immunosuppression overcomes insulin- and vector-specific immune responses that limit efficacy of AAV2/8-mediated insulin gene therapy in NOD mice. Gene Therapy, 26 (1-2), 40-56. doi:10.1038/s41434-018-0052-5
Miranda, C.J., McIntosh, J., Kia, A., Canavese, M., Foley, J., Lee, D., ...Corbau, R. (2019). Liver directed AAV gene therapy to treat Gaucher disease.
Jeyakumar, J., Kia, A., McIntosh, J., Verhoef, D., Kalcheva, P., Hosseini, P., ...Nathwani, A. (2019). Liver-directed gene therapy corrects Fabry disease in mice.
Le Quellec, S., Dane, A., Enjolras, N., McIntosh, J., Rosales, C., Negrier, C., Nathwani, A. (2019). Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins. Haemophilia, doi:10.1111/hae.13651
Recino, A., Gan, S.U., Sia, K.C., Sawyer, Y.K., Trendell, J.E., Kay, R., ...Holmes, N. (2019). Restoration of normoglycemia in diabetic models via insulin gene therapy.
Portillo, M., Kober, R., Kia, A., Dane, A.P., Sonntag, F., Horer, M., ...Nathwani, A.C. (2019). Understanding the influence of primer position in PCR based methods for titration of single stranded rAAV.

2018

Verhoef, D., Foley, J.H., Goodale, A., Macrae, E., McIntosh, J., Corbau, R., ...Nathwani, A.C. (2018). A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX.
Chowdary, P., Shapiro, S., Davidoff, A.M., Reiss, U., Alade, R., Brooks, G., ...Tuddenham, E. (2018). A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B.
Nathwani, A., Reiss, U., Tuddenham, E., Chowdary, P., McIntosh, J., Riddell, A., ...Shen, Y.M. (2018). Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer.
Paulk, N.K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., ...Haft, A. (2018). Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Molecular Therapy, 26 (1), 289-303. doi:10.1016/j.ymthe.2017.09.021
Johana, N., Tan, Y.W., Gil-Farina, I., Rosales, C., McIntosh, J., Ceiler, J., ...Nathwani, A. (2018). Comparison of Early and Late Gestation In Utero Gene Therapy, in a Non-Human Primate Model.
Marzolini, M.A.V., Akarca, A., Teng, K., Marafioti, T., Gohil, S., Nathwani, A., ...Peggs, K. (2018). Cytotoxic CD4+ Cells in Chronic Lymphocytic Leukaemia: An Extended Immunophenotypic Analysis Examining Their Association with Cytomegalovirus Serostatus and Similarities with Cytotoxic CD8+ Cells. Blood,
Harrasser, M., Gohil, S.H., Evans, R., Nathwani, A.C. (2018). Effective targeting of ROR1(+) solid tumours with next-generation Chimeric Antigen Receptor therapy.
Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., ...Nathwani, A.C. (2018). Efficacy Evaluation of Liver-Directed Gene Theraphy in Fabry Mice.
Nathwani, A., Tuddenham, E., Chowdary, P., McIntosh, J., Doyoung, L., Rosales, C., ...Meagher, M. (2018). GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant.
Brimble, M.A., Spence, Y., Zhou, J., Cheng, P., Morton, C.L., Meagher, M.M., ...Davidoff, A. (2018). In Vivo Hepatic Transfer and Transcription of AAV Producer Plasmid Contaminants.
Chan, J., Johana, N., Gil-Farina, I., Tan, Y.W., Rosales, C., Ceiler, J., ...Choolani, M. (2018). Intrauterine Gene Transfer at Early Gestation with Safe Postnatal Vector Re-Administration: A Therapeutic Strategy for Early-Onset Congenital Disease.
Gohil, S.H., Maciocia, N., Patrick, P., Roberts, T., Counsell, N., Smith, P., ...Nathwani, A.C. (2018). LenD: a study to establish the safety and efficacy of lenalidomide and dexamethasone in patients with relapsed or refractory chronic lymphocytic leukemia. Leukemia and Lymphoma, 1-4. doi:10.1080/10428194.2017.1399311
Jaffer, S., Goh, P., Abbasian, M., Nathwani, A.C. (2018). Mbd3 Promotes Reprogramming of Primary Human Fibroblast. INTERNATIONAL JOURNAL OF STEM CELLS, 11 (2), 235-241. doi:10.15283/ijsc18036
Dane, A., McIntosh, J., Lee, D., Sheridan, R., Maginn, M., Corbau, R., ...Nathwani, A.C. (2018). Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B.
Nathwani, A. (2018). PROGRESS WITH HAEMOPHILIA GENE THERAPY.
Ling, G., Nathwani, A.C., Tuddenham, E.G.D. (2018). Recent advances in developing specific therapies for haemophilia. British Journal of Haematology, doi:10.1111/bjh.15084
Recino, A., Gan, S.U., Sia, K.C., Sawyer, Y.K., Trendell, J., Kay, R., ...Holmes, N. (2018). Restoration of normoglycemia in diabetic models via insulin gene therapy.
Paul-Smith, M.C., Pytel, K.M., Gelinas, J.F., McIntosh, J., Pringle, I., Davies, L., ...Cammack, L. (2018). The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins. Gene Therapy, doi:10.1038/s41434-018-0025-8
Chan, J.K.Y., Gil-Farina, I., Johana, N., Rosales, C., Tan, Y.W., Ceiler, J., ...Schmidt, M. (2018). Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques.. FASEB Journal, fj201801391R. doi:10.1096/fj.201801391R

2017

Allsup, D., Howard, D., Munir, T., Hockaday, A., Rawstron, A., Collett, L., ...Phillips, D. (2017). A PHASE II RANDOMISED STUDY INVESTIGATING THE EFFICACY OF STANDARD OR HIGH-DOSE OFATUMUMAB IN COMBINATION WITH CHEMOTHERAPY IN RELAPSED CHRONIC LYMPHOCYTIC LEUKAEMIA.
Gohil, S., Paredes-Moscosso, S.R., Harrasser, M., El-Kholy, M., Morris, E., Nathwani, A., Della Peruta, M. (2017). A ROR1 Bispecific T Cell Engager for the Treatment of Chronic Lymphocytic Leukaemia Demonstrates Enhanced Function Following Ibrutinib Treatment.
Le Quellec, S., Dane, A.P., Negrier, C., Dargaud, Y., Nathwani, A.C., Enjolras, N. (2017). Adeno-Associated Virus Mediated Gene Transfer with Hyperfunctional FIX-E410H Variant Improves Mean Factor IX Activity Levels in Hemophilia B Mice.
Nathwani, A.C., Davidoff, A.M., Tuddenham, E.G.D. (2017). Advances in Gene Therapy for Haemophilia.. Human gene therapy, doi:10.1089/hum.2017.167
Gohil, S.H., Paredes-Moscosso, S.R., Harrasser, M., Vezzalini, M., Scarpa, A., Morris, E., ...Della Peruta, M. (2017). An ROR1 bi-specific T-cell engager provides effective targeting and cytotoxicity against a range of solid tumors. ONCOIMMUNOLOGY, 6 (7), doi:10.1080/2162402X.2017.1326437
Dane, A.P., Rosales, C., Tseng, Y., McIntosh, J.H., McKenna, R., Agbandje-McKenna, M., ...Nathwani, A.C. (2017). Analysis of the diversity of antibody response towards AAV8 capsid protein over time following a single administration of scAAV2/8-LP1-hFIXco in subjects with severe haemophilia B.
McIntosh, J.H., Kaeppel, C., Davidoff, A., Morton, C.L., Schmidt, M., Nathwani, A.C. (2017). Assessment of the integration profile of self-complementary AAV vector over time in non-human primates.
Porakishvili, N., Rajakaruna, N., Zaitseva, K., Tsertsvadze, T., Mitskevich, N., Sayed, U., ...Murphy, J. (2017). Correlation between the pattern of CD180-mediated signalling in CLL cells and changes in their surface CD180 expression in cell culture.
Allsup, D., Howard, D., Munir, T., Hockaday, A., Rawstron, A., Collett, L., ...Phillips, D. (2017). COSMIC, a phase II randomised study investigating the efficacy of standard or high-dose ofatumumab in combination with chemotherapy in patients with relapsed chronic lymphocytic leukaemia.
Paulk, N.K., Pekrun, K., Lisowski, L., Zhu, E., Nygaard, S., Li, B., ...Dane, A. (2017). Engineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Nienhuis, A.W., Nathwani, A.C., Davidoff, A.M. (2017). Gene Therapy for Hemophilia. MOLECULAR THERAPY, 25 (5), 1163-1167. doi:10.1016/j.ymthe.2017.03.033
Nathwani, A.C., Davidoff, A.M., Tuddenham, E.G.D. (2017). Gene Therapy for Hemophilia.. Hematology/oncology clinics of North America, 31 (5), 853-868. doi:10.1016/j.hoc.2017.06.011
Nathwani, A. (2017). Haemophilia gene therapy: where do we stand?.
Mattar, C.N.Z., Gil-Farina, I., Rosales, C., Johana, N., Tan, Y.Y.W., McIntosh, J., ...Choolani, M. (2017). In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. Molecular Therapy, doi:10.1016/j.ymthe.2017.04.003
Porakishvili, N., Rajakaruna, N., Sayed, U., Tsertsvadze, T., Steele, A., Mitskevich, N., ...Rai, K. (2017). Modulation of sIgM-mediated intracellular signaling pathways by CD180 toll-like receptor in Chronic Lymphocytic Leukaemia (CLL) cells.
Fernando, F., Griffin, J., Taylor, J., Camilleri, M., Lowry, L., Maraj, A., ...Gohil, S. (2017). Multicentre Retrospective Analysis of Chronic Lymphocytic Leukaemia Patients Treated with Idelalisib, "Real World" Data from the UK and Republic of Ireland - a Cohort of 68 Patients.
Gohil, S., Nathwani, A., Harrasser, M., Paredes, S., Della Peruta, M. (2017). Novel humanised ROR1 Bispecific T-cell Engager for the treatment of chronic lymphocytic leukaemia.
Gohil, S., Paredes-Moscosso, S., Harrasser, M., Davidoff, A., Pule, M., Della Peruta, M., Nathwani, A. (2017). Preclinical development of novel humanised ROR1 targeting chimeric antigen receptor T cells and bispecific T-cell engagers.
Dane, A.P., Lee, D., Zhu, C., McIntosh, J.H., Kia, A., Sonntag, F., ...Horer, M. (2017). Pre-clinical evaluation of an engineered AAV capsid designed to mediate higher transduction of human hepatocytes.
Recino, A., Foale, R., Sawyer, Y., Gan, S.U., Notaridou, M., Nichols, T., ...Lee, K.O. (2017). Restoration of normoglycaemia in diabetic models via insulin gene therapy.
Mattar, C., Biswas, A., Johana, N., Tan, Y.-.W., Rosales, C., McKintosh, J., ...Choolani, M. (2017). Safety & efficacy of intrauterine gene therapy in a clinically relevant non-human primate model.
Porakishvili, N., Sayed, U., Zaitseva, K., Rajakaruna, N., Tsertsvadze, T., Mitskevich, N., ...Rai, K.R. (2017). Synergism between CD180 and IgD-mediated phosphorylation of p38MAPK and apoptosis of p38MAPK-signaller chronic lymphocytic leukaemia (CLL) cells.
Yang, J., Milasta, S., Hu, D., AlTahan, A.M., Interiano, R.B., Zhou, J., ...Bao, J. (2017). Targeting Histone Demethylases in MYC-Driven Neuroblastomas with Ciclopirox. Cancer Research, 77 (17), 4626-4638. doi:10.1158/0008-5472.CAN-16-0826
Paul-Smith, M.C., Pytel, K.M., Gelinas, J.-.F., McIntosh, J., Pringle, I., Davies, L., ...Cameron, L. (2017). The lung as a factory to produce secreted intrapulmonary and circulatory proteins.
Post, M., Cuapio, A., Osl, M., Lehmann, D., Resch, U., Davies, D.M., ...Nathwani, A. (2017). The Transcription Factor ZnF683/hOBiT regulates human NK-cell Development. FRONTIERS IN IMMUNOLOGY, 8 doi:10.3389/fimmu.2017.00535

2016

Simeoni, I., Stephens, J.C., Hu, F., Deevi, S.V.V., Megy, K., Bariana, T.K., ...Vries, M.J.A. (2016). A high-throughput sequencing test for diagnosing inherited bleeding, thrombotic, and platelet disorders. BLOOD, 127 (23), 2791-2803. doi:10.1182/blood-2015-12-688267
Gohil, S.H., Moscosso, S.R.P., Harrasser, M., Sorio, C., Davidoff, A.M., Nathwani, A.C., Della Peruta, M. (2016). A novel bispecific T-cell engager for targeting ROR1 positive malignancies.
Gohil, S.H., Harrasser, M., Paredes-Moscosso, S.R., Nathwani, A.C., Della Peruta, M. (2016). A Novel Humanised ROR1 Bi-Specific T-Cell Engager Molecule for the Treatment of Chronic Lymphocytic Leukaemia.
Gohil, S.H., Della Peruta, M., Paredes, S., Harrasser, M., Cheung, G.W., Davies, D.M., ...Nathwani, A.C. (2016). A novel ROR1 chimeric antigen receptor for the treatment of haematological and solid malignancies.
Brimble, M.A., Zhou, J., Morton, C., Meagher, M., Nathwani, A.C., Gray, J.T., Davidoff, A.M. (2016). AAV Preparations Contain Contamination from DNA Sequences in Production Plasmids Directly Outside of the ITRs.
Davies, D.M., Paredes-Moscosso, S., Gohil, S., Della-Peruta, M., Coelho, V., Nathwani, A. (2016). Antigen density determines the efficacy of chimeric antigen receptor (CAR) re-targeted T-cells. CANCER IMMUNOLOGY RESEARCH, 4 (1), doi:10.1158/2326-6074.CRICIMTEATIAACR15-B125
Piras, B.A., Drury, J.E., Morton, C.L., Spence, Y., Lockey, T.D., Nathwani, A.C., ...Meagher, M.M. (2016). Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 3 doi:10.1038/mtm.2016.15
Piras, B.A., McNally, D.J., Lockey, T.D., Nathwani, A.C., Meagher, M.M. (2016). Evaluation of Ion-Exchange Membranes for the Purification of AAV8 from Culture Media.
Nienhuis, A.W., Nathwani, A.C., Davidoff, A.M. (2016). Gene Therapy for Hemophilia. HUMAN GENE THERAPY, 27 (4), 305-308. doi:10.1089/hum.2016.018
Davidoff, A.M., Nathwani, A.C. (2016). Genetic Targeting of the Albumin Locus to Treat Hemophilia. NEW ENGLAND JOURNAL OF MEDICINE, 374 (13), 1288-1290. doi:10.1056/NEJMcibr1600347
Hillmen, P., Ferra, C., Garcia-Marco, J., Jacob, A., Jurczak, W., Lamanna, N., ...Morchauser, F. (2016). IDELALISIB IN COMBINATION WITH BENDAMUSTINE/RITUXIMAB IMPROVES OVERALL SURVIVAL IN PATIENTS WITH RELAPSED/REFRACTORY CLL: INTERIM RESULTS OF A PHASE 3 RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED STUDY.
Mattar, C., Biswas, A., Johana, N., Tan, Y.Y.W., Rosales, C., McKintosh, J., ...Schmidt, M. (2016). Longitudinal Surveillance of Non Human Primate Recipients of Intrauterine Gene Transfer for Coagulation Factor Deficiencies.
Brimble, M.A., Reiss, U.M., Nathwani, A.C., Davidoff, A.M. (2016). Newand improved AAVenues: current status of hemophilia B gene therapy. EXPERT OPINION ON BIOLOGICAL THERAPY, 16 (1), 79-92. doi:10.1517/14712598.2015.1106475
Gohil, S.H., Della Peruta, M., Paredes-Moscosso, S.R., Harrasser, M., Cheung, G.W.-.K., Davies, D.M., ...Nathwani, A.C. (2016). Novel Humanised ROR1 Chimeric Antigen Receptors for the Treatment of Haematological Malignancies.
Paredes-Moscosso, S.R., Della Peruta, M., Gohil, S.H., Harrasser, M., Nathwani, A.C. (2016). Novel ROR1 Antibody Is Able to Trigger Specific and Superior Complement-Dependent Cytotoxicity (CDC) on Primary CLL Cells.
Nathwani, A.C., Down, J.F., Goldstone, J., Yassin, J., Dargan, P.I., Virchis, A., ...Harrison, J.D. (2016). Polonium-210 poisoning: a first-hand account. LANCET, 388 (10049), 1075-1080. doi:10.1016/S0140-6736(16)00144-6
Nathwani, A. (2016). Progress for gene therapy in haemophilia.

2015

Mattar, C., Biswas, A., Choolani, M., Johana, N., Rosales, C., McKintosh, J., ...Chan, J. (2015). A Comparison of Fetal Gene Therapy Outcomes in Early and Late Gestation.
Notaridou, M., Gan, S.U., Fu, Z.Y., Lee, K.O., Nathwani, A.C., Della Peruta, M., Calne, R.Y. (2015). Correction of murine diabetic hyperglycaemia with a single systemic administration of an AAV8 vector containing a novel codon optimized human insulin gene.
Piras, B.A., Lockey, T.D., Davidoff, A., Nienhuis, A., d'Azzo, A., Nathwani, A., Meagher, M. (2015). Distribution of AAV8 Particles in Cell Lysates and Supernatants Changes with Time and Is Dependent on the Vector Genome.
Johana, N., Tan, Y.-.W., Biswas, A., Choolani, M., Rosales, C., McKintosh, J., ...Mattar, C. (2015). Efficacy of a fetal gene therapy strategy using adeno-associated viral vectors serotypes 8 and 5 is dependent on serotype, gender of recipient and gestation: outcomes in a non-human primate model.
Nathwani, A. (2015). From Prophylaxis to Gene Therapy: How Close are We to a Cure for Hemophilia?.
Lheriteau, E., Davidoff, A.M., Nathwani, A.C. (2015). Haemophilia gene therapy: Progress and challenges. Blood Reviews, 29 (5), 321-328. doi:10.1016/j.blre.2015.03.002
Lheriteau, E., Davidoff, A.M., Nathwani, A.C. (2015). Haemophilia gene therapy: Progress and challenges. BLOOD REVIEWS, 29 (5), 321-328. doi:10.1016/j.blre.2015.03.002
Farren, T.W., Giustiniani, J., Fanous, M., Liu, F., Macey, M.G., Wright, F., ...Agrawal, S.G. (2015). Minimal residual disease detection with tumor-specific CD160 correlates with event-free survival in chronic lymphocytic leukemia. BLOOD CANCER JOURNAL, 5 doi:10.1038/bcj.2014.92
Della Peruta, M., Badar, A., Rosales, C., Chokshi, S., Kia, A., Nathwani, D., ...Davidoff, A.M. (2015). Preferential targeting of disseminated liver tumors using a recombinant adeno-associated viral vector.. HUMAN GENE THERAPY, 26 (2), 94-103. doi:10.1089/hum.2014.052
Pytel, K.M., Smith, M.P., McIntosh, J., Chan, M., Meng, C., Pringle, I., ...Hyde, S.C. (2015). Production of FVIII in the lungs.
Pytel, K.M., Paul-Smith, M., McIntosh, J., Chan, M., Meng, C., Pringle, I., ...Hyde, S.C. (2015). Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus.
Robak, T., Wach, M., Jones, J.A., Owen, C., Brown, J.R., Menter, A.R., ...Taylor, K. (2015). RESULTS OF A PHASE 3 RANDOMIZED CONTROLLED STUDY EVALUATING THE EFFICACY AND SAFETY OF IDELALISIB (IDELA) IN COMBINATION WITH OFATUMUMAB (OFA) FOR PREVIOUSLY TREATED CHRONIC LYMPHOCYTIC LEUKEMIA (CLL).
Porakishvili, N., Vispute, K., Steele, A.J., Rajakaruna, N., Kulikova, N., Tsertsvadze, T., ...Rai, K.R. (2015). Rewiring of sIgM-Mediated Intracellular Signaling through the CD180 Toll-like Receptor. MOLECULAR MEDICINE, 21 doi:10.2119/molmed.2014.00265
Porakishvili, N., Vispute, K., Steele, A.J., Rajakaruna, N., Kulikova, N., Tsertsvadze, T., ...Rai, K.R. (2015). Rewiring of sigm-mediated intracellular signaling through the CD180 toll-like receptor. Molecular Medicine, 21 46-57. doi:10.2119/molmed.2014.00265
Pytel, K.M., Paul-Smith, M.C., McIntosh, J., Chan, M., Meng, C., Pringle, I., ...Hyde, S.C. (2015). RSIV. F/HN-MEDIATED GENE THERAPY ENABLES LUNGS TO PRODUCE THERAPEUTICALLY RELEVANT LEVELS OF FVIII.
Yang, J., AlTahan, A.M., Hu, D., Wang, Y., Cheng, P.-.H., Morton, C.L., ...Fotsis, T. (2015). The Role of Histone Demethylase KDM4B in Myc Signaling in Neuroblastoma. JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE, 107 (6), doi:10.1093/jnci/djv080

2014

Awan, F.T., Hillmen, P., Hellmann, A., Robak, T., Hughes, S.G., Trone, D., ...Riveros, D. (2014). A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed chronic lymphocytic leukaemia. British Journal of Haematology, 167 (4), 466-477. doi:10.1111/bjh.13061
High, K.H., Nathwani, A., Spencer, T., Lillicrap, D. (2014). Current status of haemophilia gene therapy.. HAEMOPHILIA, 20 43-49. doi:10.1111/hae.12411
Davies, D.M., Moscosso, S.P., Gohil, S., Pule, M., Nathwani, A. (2014). Design and structural modification of ROR1 CAR T-cells as a treatment for CLL.
Della Peruta, M., Badar, A., Rosales, C., Chokshi, S., Kia, A., Nathwani, D., ...Williams, R. (2014). Development and in vivo evaluation of a liver-specific, tumour-selective recombinant adeno-associated vector to target hepatocellular carcinoma.
Dane, A., McIntosh, J., Della Peruta, M., Nathwani, A. (2014). Expression of FIX Fc fusion protein from a rAAV vector.
Chan, J., Choolani, M., Biswas, A., Johana, N., Rosales, C., McKintosh, J., ...Mattar, C. (2014). Fetal Gene Transfer in Early Gestation Promotes Stable Expression With Minimal Immune Toxicity and Facilitates Improved Expression After Postnatal Challenge.
Mattar, C., Biswas, A., Johana, N., McKintosh, J., Rosales, C., Waddington, S., ...Nathwani, A. (2014). Fetal Gene Transfer in Late Gestation Enables Stable Transgene Expression, Facilitates Postnatal Vector Re-Administration and Maintains a Good Long-Term Safety Profile.
Caxaria, S., Arthold, S., Nathwani, A.C., Goh, P.A. (2014). Generation of integration-free patient specific ips cells using episomal plasmids under feeder free conditions. 1353 355-366. doi:10.1007/7651_2015_204
Yang, C.T., French, A., Goh, P.A., Pagnamenta, A., Mettananda, S., Taylor, J., ...Watt, S.M. (2014). Human induced pluripotent stem cell derived erythroblasts can undergo definitive erythropoiesis and co-express gamma and beta globins. British Journal of Haematology, 166 (3), 435-448. doi:10.1111/bjh.12910
Yang, C.-.T., French, A., Goh, P.A., Pagnamenta, A., Mettananda, S., Taylor, J., ...Watt, S.M. (2014). Human induced pluripotent stem cell derived erythroblasts can undergo definitive erythropoiesis and co-express gamma and beta globins.. BRITISH JOURNAL OF HAEMATOLOGY, 166 (3), 435-448. doi:10.1111/bjh.12910
Nathwani, A. (2014). Long term safety and efficacy of a novel self-complementary adeno-associated viral vector encoding human FIX in patients with severe haemophilia B.
Nathwani, A.C., Reiss, U.M., Tuddenham, E.G.D., Nienhuis, A.W., Davidoff, A.M. (2014). Long term safety and efficacy of a novel self-complementary adeno-associated viral vector encoding human FIX in patients with severe haemophilia B.
Nathwani, A.C., Reiss, U.M., Tuddenham, E.G.D., Rosales, C., Chowdary, P., McIntosh, J., ...Raj, D. (2014). Long-term safety and efficacy of factor IX gene therapy in hemophilia B. NEW ENGLAND JOURNAL OF MEDICINE, 371 (21), 1994-2004. doi:10.1056/NEJMoa1407309
Nathwani, A.C., Nienhuis, A.W., Davidoff, A.M. (2014). Our journey to successful gene therapy for Hemophilia B. HUMAN GENE THERAPY, 25 (11), 923-926. doi:10.1089/hum.2014.2540
Patel, N., Nathwani, A.C., Reiss, U., Davidoff, A.M. (2014). Progress towards gene therapy for haemophilia B. INTERNATIONAL JOURNAL OF HEMATOLOGY, 99 (4), 372-376. doi:10.1007/s12185-014-1523-0
Patel, N., Reiss, U., Davidoff, A.M., Nathwani, A.C. (2014). Progress towards gene therapy for haemophilia B. International Journal of Hematology, 99 (4), 372-376. doi:10.1007/s12185-014-1523-0
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2014). Rituximab Plus Chlorambucil As First-Line Treatment for Chronic Lymphocytic Leukemia: Final Analysis of an OpenLabel Phase II Study. JOURNAL OF CLINICAL ONCOLOGY, 32 (12), 1236-+. doi:10.1200/JCO.2013.49.6547

2013

Blunt, M.D., Nathwani, A., Davies, M., Linley, A., Aguilar, M., Prentice, A., ...Forconi, F. (2013). 2-PHENYLETHYNESULFONAMIDE INDUCES THE UPR DEPENDENT APOPTOTIC PATHWAY IN CHRONIC LYMPHOCYTIC LEUKAEMIA.
Mattar, C.N., Rosales, C., Fragata, R., McIntosh, J., Johana, N., Waddington, S.N., ...Biswas, A. (2013). A robust preclinical model for fetal gene therapy: strategies targeting the liver for molecular correction of congenital single gene defects at early and late gestation. BJOG-AN INTERNATIONAL JOURNAL OF OBSTETRICS AND GYNAECOLOGY, 120 5.
Goh, P.A., Caxaria, S., Casper, C., Rosales, C., Warner, T.T., Coffey, P.J., Nathwani, A.C. (2013). A systematic evaluation of integration free reprogramming methods for deriving clinically relevant patient specific induced pluripotent stem (iPS) cells. PLoS ONE, 8 (11), doi:10.1371/journal.pone.0081622
Goh, P.A., Caxaria, S., Casper, C., Rosales, C., Warner, T.T., Coffey, P.J., Nathwani, A.C. (2013). A systematic evaluation of integration free reprogramming methods for deriving clinically relevant patient specific induced pluripotent stem (iPS) cells.. PLoS One, 8 (11), doi:10.1371/journal.pone.0081622
Nathwani, A.C., Tuddenham, E.G.D., Reiss, U.M., Recht, M., Shen, Y.-.M., Halka, K.G., ...Gray, J.T. (2013). AAV-mediated gene transfer in patients with severe hemophilia B: Longer follow-up and expansion of the high dose cohort.
Basner-Tschakarjan, E., Mingozzi, F., Chen, Y., Nathwani, A., Tuddenham, E., Riddell, A., ...Kay, M. (2013). Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector.
Cancio, M.I., Reiss, U.M., Nathwani, A.C., Davidoff, A.M., Gray, J.T. (2013). Developments in the treatment of hemophilia B: Focus on emerging gene therapy. Application of Clinical Genetics, 6 91-101. doi:10.2147/TACG.S31928
Mattar, C., Rosales, C., Fragata, R., Johana, N., Waddington, S., Biswas, A., ...Chan, J. (2013). Fetal Gene Therapy at 0.4G with AAV-hFIX or AAV-hFX Mediates Therapeutic Transgene Expression Despite Growth and a Positive Immune Response in a Non-Human Primate Model.
Mattar, C., Rosales, C., Fragata, R., Johana, N., Waddington, S., Biswas, A., ...Chan, J. (2013). Fetal Gene Therapy at Early and Late Gestation: Analysis of Outcomes and Postnatal Vector Challenge in a Macaque Model.
McIntosh, J., Lenting, P.J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., ...McVey, J.H. (2013). Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.. BLOOD, 121 (17), 3335-3344. doi:10.1182/blood-2012-10-462200
Tremblay, J.P., Xiao, X., Aartsma-Rus, A., Barbas, C., Blau, H.M., Bogdanove, A.J., ...Bueren, J.A. (2013). Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.. Molecular Therapy, 21 (2), 266-268. doi:10.1038/mt.2013.4

2012

Theodoulou, A., Berryman, J., Nathwani, A., Scully, M. (2012). Comparison of cryoprecipitate with fibrinogen concentrate for acquired hypofibrinogenaemia. Transfusion and Apheresis Science, 46 (2), 159-162. doi:10.1016/j.transci.2011.11.005
Theodoulou, A., Berryman, J., Nathwani, A., Scully, M. (2012). Comparison of cryoprecipitate with fibrinogen concentrate for acquired hypofibrinogenaemia.. TRANSFUSION AND APHERESIS SCIENCE, 46 (2), 159-162. doi:10.1016/j.transci.2011.11.005
Della Peruta, M., Binny, C., Nathwani, D., McIntosh, J., Rosales, C., Davidoff, A.M., ...Nathwani, A.C. (2012). DEVELOPMENT OF A LIVER-SPECIFIC, TUMOUR-SELECTIVE RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TO TARGET HEPATOCELLULAR CARCINOMA. GUT, 61 A201. doi:10.1136/gutjnl-2012-302514c.43
Della Peruta, M., Binny, C., Nathwani, D., McIntosh, J., Rosales, C., Davidoff, A.M., ...Nathwani, A.C. (2012). DEVELOPMENT OF A LIVER-SPECIFIC, TUMOUR-SELECTIVE RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TO TARGET HEPATOCELLULAR CARCINOMA.
Mattar, C.N., Nathwani, A., Rosales, C., Waddington, S., Johana, N., Ng, X.-.W., ...Chan, J.K.Y. (2012). Early AAV-Mediated Intrauterine Gene Therapy at 0.4G in Non-Human Primates.
Kulikova, N., Lydyard, P., Vispute, K., Steele, A., Tsertsvadze, T., Botchorishvili, E., ...Clark, E. (2012). Effect of CD180 ligation or CD180/IgM sequential ligation on signalling pathways in chronic lymphocytic leukaemia cells.
Davidoff, A., Tuddenham, E.G.D., Rangarajan, S., Rosales, C., McIntosh, J., Chowdary, P., ...Ng, C. (2012). Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B.
Nathwani, A. (2012). Stable Factor IX Activity Following AAV-mediated Gene Transfer in Patients with Severe Hemophilia B..
McIntosh, J.H., Cochrane, M., Cobbold, S., Waldmann, H., Nathwani, S.A., Davidoff, A.M., Nathwani, A.C. (2012). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Therapy, 19 (1), 78-85. doi:10.1038/gt.2011.64
McIntosh, J.H., Cochrane, M., Cobbold, S., Waldmann, H., Nathwani, S.A., Davidoff, A.M., Nathwani, A.C. (2012). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine.. GENE THERAPY, 19 (1), 78-85. doi:10.1038/gt.2011.64
Mattar, C.N., Waddington, S.N., Biswas, A., Davidoff, A.M., Choolani, M., Chan, J.K.Y., Nathwani, A.C. (2012). The case for intrauterine gene therapy. BEST PRACTICE & RESEARCH CLINICAL OBSTETRICS & GYNAECOLOGY, 26 (5), 697-709. doi:10.1016/j.bpobgyn.2012.06.006
Sack, B.K., Merchant, S., Markusic, D.M., Nathwani, A.C., Davidoff, A.M., Byrne, B.J., Herzog, R.W. (2012). Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS ONE, 7 (5), doi:10.1371/journal.pone.0037671
Binny, C.J., Nathwani, A.C. (2012). Vector systems for prenatal gene therapy: Principles of adeno-associated virus vector design and production. Methods in Molecular Biology, 891 109-131. doi:10.1007/978-1-61779-873-3_6
Binny, C.J., Nathwani, A.C. (2012). Vector systems for prenatal gene therapy: principles of adeno-associated virus vector design and production.. Methods in molecular biology (Clifton, N.J.), 891 109-131.

2011

Rosales, C., McIntosh, J., Hanley, J., Nathwani, A., Morton, C., Davidoff, A. (2011). A novel epigenetic modifier significantly improves recombinant adeno-associated virus vector mediated transgene expression in mice. HUMAN GENE THERAPY, 22 (10), A107.
Nathwani, A., Tuddenham, E.G.D., Rosales, C., Macintosh, J., Chowdary, P., Riddell, A., ...Harrington, C. (2011). A Phase I/II clinical trial entailing peripheral vein administration of a novel self complementary adeno-associated viral vector encoding human FIX for Haemophilia B gene therapy. HUMAN GENE THERAPY, 22 (10), A20.
Nathwani, A.C., Tuddenham, E.G.D., Rosales, C., Macintosh, J., Chowdary, P., Riddell, A., ...Griffioen, A. (2011). A phase i/ii clinical trial entailing peripheral vein administration of a novel self complementary adeno-associated viral vector encoding human fix for haemophilia b gene therapy.
McIntosh, J., Lenting, P., Tuddenham, E., Nawathe, S., Waddington, S., Gray, J., ...Nathwani, A. (2011). AAV mediated Factor VIII expression from a novel expression cassette with a potent codon-optimised FVIII construct. HUMAN GENE THERAPY, 22 (10), A28.
Herzog, R.W., Davidoff, A.M., Markusic, D.M., Nathwani, A.C. (2011). AAV Vector Biology in Primates: Finding the Missing Link?. Mol.Ther., 19 (11), 1923-1924. doi:10.1038/mt.2011.218
Binny, C., McIntosh, J., Peruta, M., Kymalainen, H., Tuddenham, E., Waddington, S., ...Thrasher, A. (2011). AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage. HUMAN GENE THERAPY, 22 (10), A45.
Nathwani, A., Binny, C.J., McIntosh, J.H., Tuddenham, E.G.D., Peruta, M.D., Kymalainen, H., ...Spence, Y. (2011). AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous haemorrhage.. Blood,
Nathwani, A.C., Tuddenham, E.G.D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D.C., ...Harrington, C. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.. NEW ENGLAND JOURNAL OF MEDICINE, 365 (25), 2357-2365. doi:10.1056/NEJMoa1108046
Raj, D., Peggs, K., Pule, M., Linch, D., Davies, M., Nathwani, A. (2011). Adoptive immunotherapy for patients with chronic lymphocytic leukaemia using a chimeric antigen receptor targeting CD23.
Farren, T.W., Liu, F., Macey, M.G., Jenner, M., Prentice, A., Nathwani, A., Agrawal, S.G. (2011). CD160: a novel approach to minimal residual disease (MRD) assessment in chronic lymphocytic leukaemia (CLL).
Porakishvili, N., Memon, A., Vispute, K., Kulikova, N., Clark, E.A., Rai, K.R., ...Lydyard, P.M. (2011). CD180 functions in activation, survival and cycling of B chronic lymphocytic leukaemia cells.. BRITISH JOURNAL OF HAEMATOLOGY, 153 (4), 486-498. doi:10.1111/j.1365-2141.2011.08605.x
Ward, N.J., Buckley, S.M.K., Waddington, S.N., Vandendriessche, T., Chuah, M.K.L., Nathwani, A.C., ...Thrasher, A.J. (2011). Codon optimization of human factor VIII cDNAs leads to high-level expression.. BLOOD, 117 (3), 798-807. doi:10.1182/blood-2010-05-282707
Denbo, J.W., Williams, R.F., Orr, W.S., Sims, T.L., Ng, C.Y., Zhou, J., ...Duntsch, C. (2011). Continuous local delivery of interferon-beta stabilizes tumor vasculature in an orthotopic glioblastoma xenograft resection model. Surgery, 150 (3), 497-504. doi:10.1016/j.surg.2011.07.044
Mingozzi, F., Basner-Tschakarjan, E., Chen, Y., Nathwani, A.C., Tuddenham, E.G.D., Rosales, C., ...Glader, B. (2011). Dose-dependent activation of capsid-specific T cells after AAV serotype 8 vector administration in a clinical study for hemophilia B. JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 9 761-762.
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2011). Final response assessment of the CLL208 trial: an open-label phase II study to investigate the safety and efficacy of rituximab plus chlorambucil in previously untreated patients with CD20-positive B-cell chronic lymphocytic leukaemia (CLL)..
Casper, C.J., Warner, T.T., Taanman, J.W., Nathwani, A.C. (2011). Generation of iPS-derived dopaminergic neurons as an in-vitro model of Rapid-Onset Dystonia Parkinsonism. HUMAN GENE THERAPY, 22 (10), A42-A43.
Allay, J.A., Sleep, S., Long, S., Tillman, D.M., Clark, R., Carney, G., ...Davidoff, A.M. (2011). Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial. HUMAN GENE THERAPY, 22 (5), 595-604. doi:10.1089/hum.2010.202
Rahim, A.A., Wong, A.M., Ahmadi, S., Hoefer, K., Buckley, S.M., Hughes, D.A., ...Cooper, J.D. (2011). In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression. Gene Ther., doi:10.1038/gt.2011.157
Nathwani, A.C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., ...Jackson, S. (2011). Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol.Ther., 19 (5), 876-885. doi:10.1038/mt.2010.274
David, A.L., McIntosh, J., Peebles, D.M., Cook, T., Waddington, S., Weisz, B., ...Davidoff, A.M. (2011). Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.. HUMAN GENE THERAPY, 22 (4), 419-426. doi:10.1089/hum.2010.007
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2011). RITUXIMAB PLUS CHLORAMBUCIL (R-CHLORAMBUCIL) AS FIRST-LINE TREATMENT FOR CHRONIC LYMPHOCYTIC LEUKAEMIA (CLL): FINAL ANALYSIS OF AN OPEN-LABEL PHASE II STUDY.
Raj, D., Davidoff, A.M., Nathwani, A.C. (2011). Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.. EXPERT REVIEW OF HEMATOLOGY, 4 (5), 539-549. doi:10.1586/EHM.11.48
Mattar, C.N., Nathwani, A.C., Waddington, S.N., Dighe, N., Kaeppel, C., Nowrouzi, A., ...Fisk, N.M. (2011). Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques. Mol.Ther., 19 (11), 1950-1960. doi:10.1038/mt.2011.107
McIntosh, J.H., Cochrane, M., Cobbold, S., Waldmann, H., Nathwani, S.A., Davidoff, A.M., Nathwani, A.C. (2011). Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Ther., doi:10.1038/gt.2011.64
Fagone, P., Wright, J.F., Nathwani, A.C., Nienhuis, A.W., Davidoff, A.M., Gray, J.T. (2011). Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods. Hum.Gene Ther., doi:10.1089/hum.2011.104
Binny, C., Della Peruta, M., Nathwani, A.C. (2011). Targeted gene therapy for hepatocellular carcinoma: A reality?. In Clinical Dilemmas in Primary Liver Cancer. (pp. 184-190). .
Della Peruta, M., Binny, C., Nathwani, D., Chokshi, S., McIntosh, J., Rosales, C., ...Nathwani, A.C. (2011). The development of a tumour-selective recombinant adeno-associated virus vector to target hepatocellular carcinoma. HUMAN GENE THERAPY, 22 (10), A113.

2010

Georgiadis, A., Tschernutter, M., Bainbridge, J.W., Robbie, S.J., McIntosh, J., Nathwani, A.C., ...Ali, R.R. (2010). AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins. Gene Ther., 17 (4), 486-493. doi:10.1038/gt.2009.162
Hanley, J., Rastegarlari, G., Nathwani, A.C. (2010). An introduction to induced pluripotent stem cells. Br.J.Haematol., 151 (1), 16-24. doi:10.1111/j.1365-2141.2010.08296.x
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D.W., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2010). An open-label phase II study to investigate the safety and efficacy of rituximab plus chlorambucil in previously untreated patients with CD20-positive B-cell chronic lymphocytic leukaemia (CLL): On behalf of the NCRI CLL sub-group.
Binny, C.J., McIntosh, J.H., Tuddenham, E., Waddington, S., Buckley, S., Thrasher, A., ...Davidoff, A. (2010). Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System.
Binny, C.J., McIntosh, J.H., Tuddenham, E., Waddington, S., Buckley, S., Thrasher, A., ...Davidoff, A. (2010). Assessing the Potential of Perinatal Gene Transfer Using Congenital Factor VII Deficiency as a Model System.
Porakishvili, N., Lydyard, P., Bremser, A., Vispute, K., Memon, A., Kulikova, N., ...Damle, R.N. (2010). B Cell Chronic Lymphocytic Leukemia (B CLL) Cells Unresponsive to CD180 Ligation Fail to Respond to Anti IgM Stimulation as Well.
Porakishvili, N., Lydyard, P., Bremser, A., Vispute, K., Memon, A., Kulikova, N., ...Damle, R.N. (2010). B-Cell Chronic Lymphocytic Leukemia (B-CLL) Cells Unresponsive to CD180 Ligation Fail to Respond to Anti-IgM Stimulation as Well.
Ward, N.J., Buckley, S.M., Waddington, S.N., Nathwani, A.C., Tuddenham, E.G., Kinnon, C., ...McVey, J.H. (2010). Correction of Murine Hemophilia A Using SIN Lentiviral Vectors Expressing Enhanced Human Factor VIII cDNAs.
Nathwani, A., Tuddenham, E., Rosales, C., McIntosh, J., Riddell, A., Rustagi, P., ...Coleman, J. (2010). Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B.
Nathwani, A., Tuddenham, E., Rosales, C., McIntosh, J., Riddell, A., Rustagi, P., ...Coleman, J. (2010). Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B.
Nathwani, A.C., Tuddenham, E.G.D., Rosales, C., McIntosh, J., Riddell, A., Rustagi, P., ...Coleman, J. (2010). Early clinical trial results following administration of a low dose of a novel self complementary adenohyphen;associated viral vector encoding human factor ix in two subjects with severe Haemophilia B.
Tan, M.H., Smith, A.J., Pawlyk, B., Xu, X., Liu, X., Bainbridge, J.B., ...Nathwani, A. (2010). Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors (vol 18, pg 2099, 2009). HUM MOL GENET, 19 (4), 735. doi:10.1093/hmg/ddp540
Nathwani, A. (2010). Haemophilia B gene therapy study in the UK. HAEMOPHILIA, 16 (6), 973.
Sims, T.L., McGee, M., Williams, R.F., Myers, A.L., Tracey, L., Hamner, J.B., ...McCarville, B. (2010). IFN-beta restricts tumor growth and sensitizes alveolar rhabdomyosarcoma to ionizing radiation. Mol.Cancer Ther., 9 (3), 761-771. doi:10.1158/1535-7163.MCT-09-0800
Sims, T.L., McGee, M., Williams, R.F., Myers, A.L., Tracey, L., Hamner, J.B., ...McCarville, B. (2010). IFN-β restricts tumor growth and sensitizes alveolar rhabdomyosarcoma to ionizing radiation. Molecular Cancer Therapeutics, 9 (3), 761-771. doi:10.1158/1535-7163.MCT-09-0800
McGee, M.C., Hamner, J.B., Williams, R.F., Rosati, S.F., Sims, T.L., Ng, C.Y., ...Nathwani, A.C. (2010). Improved intratumoral oxygenation through vascular normalization increases glioma sensitivity to ionizing radiation. Int.J.Radiat.Oncol.Biol.Phys., 76 (5), 1537-1545. doi:10.1016/j.ijrobp.2009.12.010
Farren, T., Liu, F.T., Stephens, C., Macey, M., Jenner, M., Prentice, A., ...Agrawal, S. (2010). Inter Laboratory Assessment of Minimal Residual Disease In Chronic Lymphocytic Leukemia - Evaluation of the Novel CD160FCA Assay.
Farren, T., Liu, F., Stephens, C., Macey, M., Jenner, M., Prentice, A., ...Agrawal, S. (2010). Inter-Laboratory Assessment of Minimal Residual Disease In Chronic Lymphocytic Leukemia-7 Evaluation of the Novel CD160FCA Assay.
Mattar, C.N., AC, N., Dighe, N., Waddington, S., McIntosh, J., Biswas, A., ...Peebles, D. (2010). Late-Gestation Intravascular Delivery of Self-Complementary AAV Leads to Sustained Transgene Expression with the Possibility of Vector Integration Events in the Liver.
Williams, R.F., Sims, T.L., Tracey, L., Myers, A.L., Ng, C.Y., Poppleton, H., ...Davidoff, A.M. (2010). Maturation of tumor vasculature by interferon-beta disrupts the vascular niche of glioma stem cells. Anticancer Res., 30 (9), 3301-3308.
Tracey, L., Streck, C.J., Du, Z., Williams, R.F., Pfeffer, L.M., Nathwani, A.C., Davidoff, A.M. (2010). NF-kappa B activation mediates resistance to IFN beta in MLL-rearranged acute lymphoblastic leukemia. LEUKEMIA, 24 (4), 806-812. doi:10.1038/leu.2010.2
Tracey, L., Streck, C.J., Du, Z., Williams, R.F., Pfeffer, L.M., Nathwani, A.C., Davidoff, A.M. (2010). NF-κB activation mediates resistance to IFNΒ in MLL-rearranged acute lymphoblastic leukemia. Leukemia, 24 (4), 806-812. doi:10.1038/leu.2010.2
Walton, J.A., Lydyard, P.M., Nathwani, A., Emery, V., Akbar, A., Glennie, M.J., Porakishvili, N. (2010). Patients with B cell chronic lymphocytic leukaemia have an expanded population of CD4 perforin expressing T cells enriched for human cytomegalovirus specificity and an effector-memory phenotype.. Br J Haematol, 148 (2), 274-284. doi:10.1111/j.1365-2141.2009.07964.x
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2010). Rituximab Plus Chlorambucil In Patients with CD20-Positive B Cell Chronic Lymphocytic Leukemia (CLL) Final Response Analysis of An Open Label Phase H Study.
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2010). Rituximab Plus Chlorambucil In Patients with CD20-Positive B-Cell Chronic Lymphocytic Leukemia (CLL): Final Response Analysis of An Open-Label Phase II Study.
McIntosh, J., Lenting, P.J., Tuddenham, E., Sotannde, M., Waddington, S., Gray, J.T., ...Nathwani, A. (2010). Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant.
McIntosh, J., Lenting, P.J., Tuddenham, E., Sotannde, M., Waddington, S., Gray, J.T., ...Nathwani, A. (2010). Stable High Level Coagulation Factor VIII Expression In Vivo Following Gene Transfer Using a Novel Expression Cassette Encoding a More Potent FVIII Variant.
Williams, R.F., Myers, A.L., Sims, T.L., Ng, C.Y., Nathwani, A.C., Davidoff, A.M. (2010). Targeting multiple angiogenic pathways for the treatment of neuroblastoma. J.Pediatr.Surg., 45 (6), 1103-1109. doi:10.1016/j.jpedsurg.2010.02.073

2009

Shih, C.S., Laurie, N., Holzmacher, J., Spence, Y., Nathwani, A.C., Davidoff, A.M., Dyer, M.A. (2009). AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models. Neuromolecular.Med., 11 (1), 43-52. doi:10.1007/s12017-009-8059-0
Hillmen, P., Gribben, J.G., Follows, G.A., Milligan, D.W., Sayala, H.A., Moreton, P., ...Pettitt, A.R. (2009). An Open-Label Phase II Study to Investigate the Safety and Efficacy of Rituximab Plus Chlorambucil in Previously Untreated Patients with CD20-Positive B-Cell Chronic Lymphocytic Leukaemia (CLL)..
Reiss, U., Davidoff, A., Gray, J., Nienhuis, A., Nathwani, A. (2009). Dose response results of self complementary adeno-associated virus (AAV) vector-mediated factor IX gene transfer in non-human primates. HAEMOPHILIA, 15 (2), 635. doi:10.1111/j.1365-2516.2009.01996_6.x
Nathwani, A.C., Cochrane, M., McIntosh, J., Ng, C.Y.C., Zhou, J., Gray, J.T., Davidoff, A.M. (2009). Enhancing transduction of the liver by adeno-associated viral vectors. GENE THERAPY, 16 (1), 60-69. doi:10.1038/gt.2008.137
Kirwan, M., Beswick, R., Vulliamy, T., Nathwani, A.C., Walne, A.J., Casimir, C., Dokal, I. (2009). Exogenous TERC alone can enhance proliferative potential, telomerase activity and telomere length in lymphocytes from dyskeratosis congenita patients. Br.J.Haematol., 144 (5), 771-781. doi:10.1111/j.1365-2141.2008.07516.x
Mattar, C.N., Dighe, N.M., Waddington, S.N., McIntosh, J., Biswas, A., Fisk, N.M., ...Chan, J. (2009). Long-Term Transgene Expression in New-Born Cynomolgus Macaques Following Intra-Uterine Gene Transfer of Self-Complementary AAV.
Ward, N.J., Apolonia, L., Cochrane, M., Buckley, S.M., Waddington, S.N., Nathwani, A.C., ...Thrasher, A.J. (2009). Nonintegrating Lentiviral Vectors for Treatment of Haemophilia B.

2008

Natkunarajah, M., Trittibach, P., McIntosh, J., Duran, Y., Barker, S.E., Smith, A.J., ...Ali, R.R. (2008). Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Therapy, 15 (6), 463-467.
Carlisle, R.C., Benjamin, R., Briggs, S.S., Sumner-Jones, S., McIntosh, J., Gill, D., ...Ulbrich, K. (2008). Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera. The Journal of Gene Medicine, 10 (4), 400-411.
Nathwani, A., Quinn, J.P., Mohamedbhai, S., Chipperfield, K., Treacy, M., D'Sa, S. (2008). Efficacy of rituximab in combination with steroids in refractory chronic lymphocytic leukemia.. Leuk Lymphoma, 49 (10), 1995-1998.
Kyriakou, C., Rabin, N., Pizzey, A., Nathwani, A., Yong, K. (2008). Factors that influence short-term homing of human bone marrow-derived mesenchymal stem cells in a xenogeneic animal model. Haematologica, 93 (10), 1457-1465.
Nathwani, A., Rosati, S.F., Williams, R.F., Nunnally, L.C., McGee, M.C., Sims, T.L., ...Ng, C.Y. (2008). IFN-beta sensitizes neuroblastoma to the antitumor activity of temozolomide by modulating O6-methylguanine DNA methyltransferase expression.. Mol Cancer Ther, 7 (12), 3852-3858.
Rosati, S.F., Williams, R.F., Nunnally, L.C., McGee, M.C., Sims, T.L., Tracey, L., ...Nathwani, A.C. (2008). IFN-β sensitizes neuroblastoma to the antitumor activity of temozolomide by modulating O⁶-methylguanine DNA methyltransferase expression. Molecular Cancer Therapeutics, 7 (12), 3852-3858. doi:10.1158/1535-7163.MCT-08-0806
David, A.L., McIntosh, J., Nathwani, A., Cook, T., Boyd, M., Weisz, B., ...Peebles, D. (2008). Long term expression of human factor IX after ultrasound-guided fetal gene therapy in sheep.
David, A.L., McIntosh, J., Weisz, B., Boyd, M., Cook, T., Wigley, V., ...Peebles, D.M. (2008). Long term perinatal gene transfer after clinically applicable delivery of prenatal gene therapy in the sheep.
Evans, A., Riva, A., Cooksley, H., Phillips, S., Nathwani, A., Craigen, J., ...Naoumov, N. (2008). Longitudinal analysis of PD1 expression: Impact of HBeAg seroconversion during antiviral treatment of chronic hepatitis B.
David, A.L., McIntosh, J., Nathwani, A., Cook, T., Boyd, M., Weisz, B., ...Peebles, D.M. (2008). Long-term expression of human factor IX after ultrasound-guided delivery of AAV8 hFIX to fetal sheep in utero.
Buckley, S.M., Howe, S.J., Rahim, A.A., Buning, H., McIntosh, J., Wong, S.P., ...Coutelle, C. (2008). Luciferin Detection After Intranasal Vector Delivery Is Improved by Intranasal Rather Than Intraperitoneal Luciferin Administration.. Hum.Gene Ther., 19 (10), 1050-1056.
Buckley, S.M., Howe, S.J., Wong, S.P., Buning, H., McIntosh, J., Baker, A., ...McKay, T.R. (2008). Luciferin detection after intra-nasal vector delivery is improved by intra-nasal rather than intra-peritoneal luciferin administration.. Hum Gene Ther, doi:10.1089/hgt.2008.023
Ward, N.J., Apolonia, L., Fernandes, C., Waddington, S.N., Cochrane, M., Nathwani, A.C., ...Philpott, N.J. (2008). Non-integrating lentiviral vector pseudotypes for safer gene therapy.
Graham, T., McIntosh, J., Work, L.M., Nathwani, A., Baker, A.H. (2008). Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats.. Genetic vaccines and therapy, 6 9-. doi:10.1186/1479-0556-6-9
Graham, T., McIntosh, J., Work, L.M., Nathwani, A., Baker, A.H. (2008). Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats. Genetic Vaccines and Therapy, 3 6-9.
Osman, E., Evans, V., Graham, I.R., Athanasopoulos, T., McIntosh, J., Nathwani, A.C., ...J, S. (2008). Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice. Atherosclerosis, 204 (1), 121-126. doi:10.1016/j.atherosclerosis.2008.08.043
Evans, A., Riva, A., Cooksley, H., Phillips, S., Puranik, S., Nathwani, A., ...Naoumov, N.V. (2008). Programmed death 1 expression during antiviral treatment of chronic hepatitis B: Impact of hepatitis B e-antigen seroconversion. Hepatology, 48 (3), 759-769.
Buch, P.K., McIntosh, J., Georgiadis, A., Mowat, F., Tschernutter, M., Smith, A.J., ...Ali, R.R. (2008). Pseudotyped adeno-associated viral vectors to enhance gene replacement therapy in a mouse model of retinal degeneration.
Quinn, J., Mohamedbhai, S., Treacy, M., D'Sa, S., Nathwani, A. (2008). Rituximab in combination with high-dose steroids: a safe and effective salvage regimen in advanced, refractory chronic lymphocytic leukaemia.
Kyriakou, C., Rabin, N., Pizzey, A., Nathwani, A., Yong, K. (2008). Short term homing of human bone marrow derived mesenchymal stem cells (hMSCs) in a xenogeneic animal model is influenced by animal age and is increased by enforced expression of CXCR4. Haematologica, 93 (10), 1457-1465.
Delgado, J., Pillai, S., Benjamin, R., Caballero, D., Martino, R., Nathwani, A., ...Sureda, A. (2008). The effect of in vivo T cell depletion with alemtuzumab on reduced-intensity allogeneic hematopoietic cell transplantation for chronic lymphocytic leukemia.. Biology of Blood and Marrow Transplantation, 14 (11), 1288-1297.

2007

Rabin, N., Kyriakou, C., Coulton, L., Gallagher, O.M., Buckle, C., Benjamin, R., ...Nathwani, A.C. (2007). A new xenograft model of myeloma bone disease demonstrating the efficacy of human mesenchymal stem cells expressing osteoprotegerin by lentiviral gene transfer.. Leukemia,
Riva, A., Paulon, E., Nathwani, A., Taylor, J., Naoumov, N.V. (2007). Adenovirus-mediated delivery of HBV DNA in dendritic cells: A potent approach to overcome impaired T-cell reactivity in chronic hepatitis B.
Barkley, L.R., Hong, H.K., Kingsbury, S.R., McCormick, L.A., James, M., Nathwani, A.C., ...Williams, G.H. (2007). Cdc6 controls proliferative capacity during establishment of the differentiated state. Experimental Cell Research, 313 (17), 3789-3799. doi:10.1016/j.yexcr.2007.07.004
Benjamin, R., Singh, N., McIntosh, J., Meager, A., Wadhwa, M., Streck, C., ...Nathwani, A.C. (2007). Continuous delivery of human type I interferons ({alpha}/{beta}) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model.. Blood, 109 (3), 1244-1247.
Benjamin, R., Khwaja, A., Singh, N., McIntosh, J., Meager, A., Wadhwa, M., ...Nathwan, A.C. (2007). Continuous delivery of human type I interferons (α/β)has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model. Blood, 109 (3), 1244-1247.
Nathwani, A., Dickson, P.V., Hamner, J.B., Streck, C.J., Ng, C.Y., McCarville, M.B., ...Wilson, C.M. (2007). Continuous delivery of IFN-beta promotes sustained maturation of intratumoral vasculature.. Mol Cancer Res, 5 (6), 531-542.
Vaessen, S.F.C., Comijn, L., Snapper, J., Nathwani, A.C., Kastelein, J., Petry, H., ...Twisk, J. (2007). Correction of HDL deficiency in ApoA-I-deficient mice following self-complementary AAV-mediated liver expression of human ApoA-I.
Osman, E., Graham, I., Athanasopoulos, T., Nathwani, A.C., Owen, J.S. (2007). Liver-directed gene transfer of atheroprotective human ApoA-I(Milano).
Rabin, N., Coulton, L., Gallagher, O.M., Benjamin, R., Singh, N., McIntosh, J.H., ...Yong, K. (2007). Micro-computer tomography (MICROCT) analysis demonstrates extensive bone loss in the KMS-12-BM xenogeneic murine model of myeloma.
Ward, N., Apolonia, L., Fernandes, C., Waddington, S., Cochrane, M., Kinnon, C., ...Philpott, N. (2007). Non-integrating lentiviral vector pseudotypes for safer gene therapy.
Quinn, J., Mohamedbhai, S., Treacey, M., D'Sa, S., Nathwani, A. (2007). Rituximab in combination with high-dose steroids in advanced, refractory chronic lymphocytic leukaemia.
Nathwani, A.C., Gray, J.T., McIntosh, J., Ng, C.Y., Zhou, J., Spence, Y., ...Davidoff, A.M. (2007). Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.. Blood, 109 (4), 1414-1421. doi:10.1182/blood-2006-03-010181

2006

McCarville, M.B., Streck, C.J., Dickson, P.V., Li, C.S., Nathwani, A.C., Davidoff, A.M. (2006). Angiogenesis inhibitors in a murine neuroblastoma model: Quantitative assessment of intratumoral blood flow with contrast-enhanced gray-scale US. Radiology, 240 (1), 73-81. doi:10.1148/radiol.2401050709
McCarville, M.B., Streck, C.J., Dickson, P.V., Li, C.S., Nathwani, A.C., Davidoff, A.M. (2006). Angiogenesis inhibitors in a murine neuroblastoma model: quantitative assessment of intratumoral blood flow with contrast-enhanced gray-scale US.. Radiology, 240 (1), 73-81.
Streck, C.J., Dickson, P.V., Ng, C.Y., Zhou, J., Hall, M.M., Gray, J.T., ...Davidoff, A.M. (2006). Antitumor efficacy of AAV-mediated systemic delivery of interferon-beta.. Cancer Gene Therapy, 13 (1), 99-106.
Mcintosh, J., Cochrane, M., Tuddenham, E.G.D., Davidoff, A.M., Nathwani, A.C. (2006). Expression of human FIX at therapeutic levels in nonhuman primates following a single peripheral vein infusion of low doses of self complementary AAV vector renews hope for haemophilia B gene therapy.
Kyriakou, C., Rabin, N., Nathwani, A., Pizzey, A., Linch, D.C., Yong, K. (2006). Factors influencing short term in vivo biodistribution of human bone marrow derived mesenchymal stem cells (hMSCs) in a xenogeneic animal model..
Rabin, N., Kyriakou, C., Benjamin, R., Pizzey, A., Gallagher, O., Singh, N., ...Yong, K. (2006). Gene modified human mesenchymal stem cells expressing osteoprotegerin inhibit osteoclast activation and increase trabecular bone area in a xenogeneic murine model of myeloma..
Rabin, N., Kyriakou, C., Pizzey, A., Buckle, C., Prideaux, M., Croucher, P., ...Yong, K. (2006). Gene Modified Human Mesenchymal Stem Cells Expressing Osteoprotegerin Reverse Osteoclast Activation in a Xenogeneic Murine Model of Multiple Myeloma. MOLECULAR THERAPY, 13 S256. doi:10.1016/j.ymthe.2006.08.740
Kyriakou, C.A., Yong, K.L., Benjamin, R., Pizzey, A., Dogan, A., Singh, N., ...Nathwani, A.C. (2006). Human mesenchymal stem cells (hMSCs) expressing truncated soluble vascular endothelial growth factor receptor (tsFlk-1) following lentiviral-mediated gene transfer inhibit growth of Burkitt's lymphoma in a murine model.. The Journal of Gene Medicine, 8 (3), 253-264.
Nathwani, A.C., Gray, J.T., Ng, C.Y., Zhou, J., Spence, Y., Waddington, S.N., ...Boon-Spijker, M. (2006). Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.. Blood, 107 (7), 2653-2661.
McIntosh, J.H., Cochrane, M., Prizzey, A., Waldmann, H., Davidoff, A.M., Nathwani, A.C. (2006). Successful Readministration of AAV8 Following Administration of Nondepleting CD4 Antibody at the Time of Vector Administration. MOLECULAR THERAPY, 13 S190. doi:10.1016/j.ymthe.2006.08.560
Benjamin, R., Davidoff, A.M., Singh, N., Mcintosh, J., Khwaja, A., Streck, C., ...Nathwani, A.C. (2006). Therapeutic efficacy of interferon-beta in a xenograft model of acute myeloid leukaemia.

2005

Nathwani, A.C., Davidoff, A.M., Linch, D.C. (2005). A review of gene therapy for haematological disorders.. British Journal of Haematology, 128 (1), 3-17.
Nathwani, A.C., Davidoff, A.M., Linch, D.C. (2005). A review of gene therapy for haematological disorders.. British journal of haematology, 128 (1), 3-17. doi:10.1111/j.1365-2141.2004.05231.x
Streck, C.J., Zhang, Y., Zhou, J., Ng, C., Nathwani, A.C., Davidoff, A.M. (2005). Adeno-associated virus vector-mediated delivery of pigment epithelium-derived factor restricts neuroblastoma angiogenesis and growth.. Journal of Pediatric Surgery, 40 (1), 236-243.
Streck, C.J., Dickson, P.V., Ng, C.Y., Zhou, J., Gray, J.T., Nathwani, A.C., Davidoff, A.M. (2005). Adeno-associated virus vector-mediated systemic delivery of IFN-beta combined with low-dose cyclophosphamide affects tumor regression in murine neuroblastoma models.. Clinical Cancer Research, 11 (16), 6020-6029.
Nathwani, A.C., McIntosh, J., Davidoff, A.M. (2005). An update on gene therapy for hemophilia.. Current Hematology Reports, 4 (4), 287-293.
Nathwani, A.C., McIntosh, J., Davidoff, A.M. (2005). An update on gene therapy for hemophilia.. Current hematology reports, 4 (4), 287-293.
Davidoff, A.M., Ng, C.Y., Zhang, Y., Streck, C., Mabry, S., Barton, S., ...Vanin, E. (2005). Careful decoy receptor titering is required to inhibit tumor angiogenesis while avoiding adversely altering VEGF bioavailability.. Molecular Therapy, 11 (2), 300-310. doi:10.1016/j.ymthe.2004.09.014
Porakishvili, N., Kulikova, N., Jewell, A.P., Youinou, P.Y., Nedelec, S., Yong, K., ...Hamblin, T.J. (2005). CD180 is more highly expressed by B cell chronic lymphocytic leukaemia (B-CLL) cells using IgVh mutated than those using IgVh unmutated genes. British Journal of Haematology, 131 (3), 313-319.
Walton, J., Lawson, K., Manoussaka, M.S., Nathwani, A., Emory, V., Lydyard, P.M., Porakishvili, N. (2005). CD4(+) perforin(+) T cells in B-CLL: MHC class II restriction and anti-cytomegalovirus reactivity..
Davidoff, A.M., Gray, J.T., Ng, C.Y., Zhang, Y., Zhou, J., Spence, Y., ...Nathwani, A.C. (2005). Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models.. Molecular Therapy, 11 (6), 875-888.
Jewell, A.P., Cochrane, M., McIntosh, J., Benjamin, R., Nathwani, A. (2005). Comparison of viral vectors for gene transfer into CLL cells: Efficient transduction with adeno-associated virus-8 (AAV-8)..
Dickson, P.V., Nathwani, A.C., Davidoff, A.M. (2005). Delivery of antiangiogenic agents for cancer gene therapy.. Technology in Cancer Research and Treatment, 4 (4), 331-341.
Streck, C.J., Ng, C.Y., Zhang, Y., Zhou, J., Nathwani, A.C., Davidoff, A.M. (2005). Interferon-mediated anti-angiogenic therapy for neuroblastoma.. Cancer Letters, 228 (1), 163-170.
Manoussaka, M.S., Memon, A., Nathwani, A., Porakishvili, N., Clark, E., Lydyard, P.M. (2005). Toll like receptors (TLRs) in B cell chronic lymphocytic leukemia (B-CLL)..

2004

Gray, J.T., Davidoff, A.M., Nathwani, A.C. (2004). A novel human factor IX expression cassette packaged as self-complementary DNA dimers in adeno-associated virus vectors results in dramatically improved liver transduction, significantly improving prospects for hemophilia B gene therapy.
Porakishvili, N., Manoussaka, M., Kulikova, N., Walton, J., Nathwani, A., Jewell, A.P., ...Lydyard, P.M. (2004). Activation and proliferation of B cell chronic lymphocytic leukaemia (B-CLL) cells through anti-CD180, Anti-CD40 and IL-4..
Davidoff, A.M., Nathwani, A.C. (2004). Antiangiogenic gene therapy for cancer treatment.. Current Hematology Reports, 3 (4), 267-273.
Nathwani, A.C., Benjamin, R., Nienhuis, A.W., Davidoff, A.M. (2004). Current status and prospects for gene therapy. VOX SANGUINIS, 87 (2), 73-81. doi:10.1111/j.1423-0410.2004.00543.x
Porakishvili, N., Kulikova, N., Manoussaka, M., Jewell, A.P., Youinou, P.Y., Nedelec, S., ...Hamblin, T. (2004). Differential expression of CD180 and sIgM by B chronic lymphocytic leukaemia cells (B-CLL) using mutated and unmutated IgVh genes..
Streck, C.J., Zhou, J., Ng, C.Y., Zhang, Y., Nathwani, A.C., Davidoff, A.M. (2004). Longterm recombinant adeno-associated, virus-mediated, liver-generated expression of an angiogenesis inhibitor improves survival in mice with disseminated neuroblastoma.. Journal of the American College of Surgeons, 199 (1), 78-86.
Walton, J., Lawson, K., Manoussaka, M.S., Nathwani, A., Emory, V., Lydyard, P.M., Porakishvili, N. (2004). Perforin expressing CD4+T cells respond to CMV antigens in CMV seropositive patients with B cell chronic lymphocytic leukaemia (B-CLL)..
Nathwani, A.C., Davidoff, A.M., Tuddenham, E.G. (2004). Prospects for gene therapy of haemophilia.. Haemophilia, 10 (4), 309-318.
Davidoff, A.M., Ng, C.Y.C., Sleep, S., Gray, J., Azam, S., Zhao, Y., ...Nathwani, A.C. (2004). Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. JOURNAL OF VIROLOGICAL METHODS, 121 (2), 209-215. doi:10.1016/j.jviromet.2004.07.001
Streck, C.J., Zhang, Y., Miyamoto, R., Zhou, J., Ng, C.Y.C., Nathwani, A.C., Davidoff, A.M. (2004). Restriction of neuroblastoma angiogenesis and growth by interferon-alpha/beta.
Streck, C.J., Zhang, Y., Miyamoto, R., Zhou, J., Ng, C.Y., Nathwani, A.C., Davidoff, A.M. (2004). Restriction of neuroblastoma angiogenesis and growth by interferon-alpha/beta.. Surgery, 136 (2), 183-189.
Lajmi, A.R., Sleep, S., Ng, C., Zhou, J.F., Spence, Y.Y., Davidoff, A.M., Nathwani, A.C. (2004). Scaleable membrane chromatography purification method for adeno-associated viral vectors..
Nivsarkar, M.S., Nathwani, A., Mosley, K., Davies, N., Gregory, L.G., David, A.L., ...Waddington, S.N. (2004). Testosterone induced increase in transgene expression in vivo using Adenoviral Gene Delivery Vectors.
Folarin, N.I., Baker, R.J., Duke, V., Yogashangary, B.C., Vadikolia, C., Wickremasinge, R.G., ...Nathwani, A. (2004). Zap-70 and IgVH mutation status in combination with p53 functional analysis identifies the response of poor prognosis B-CLL patients to conventional cytotoxic agents.

2003

Porakishvili, N., Slovick, A., Jewell, A.P., Youinou, P.Y., Nathwani, A., Yong, K., ...Lydyard, P.M. (2003). Activation of B-CLL cells by anti CD180 monoclonal antibodies: Responder and non-responder patients..
McCormick, A.L., Benjamin, R., Pizzey, A., Kyriakou, C., Nathwani, A.C. (2003). Facilitation of lentivirus mediated gene transfer into primary human and murine progenitors by Antp peptide.
Ng, C.Y., Karimipoor, M., Zhou, J.F., Lajmi, A., Zhong, W.Y., Spence, Y., ...Nathwani, A.C. (2003). Highly pure recombinant adeno-associated viral (rAAV) vectors based on serotype 8 generated by a simple scalable chromatography method efficiently transduce murine liver..
Kyriakou, C.A., Yong, K.L., Benjamin, R., Gandini, D., Davidoff, A.M., Nathwani, A.C. (2003). Human mesenchymal stem cells (hMSCs) expressing truncated soluble vascular endothelial growth factor receptor (tsFlk-1) following lentiviral mediated gene transfer inhibit growth of Burkitt's lymphoma in murine models..
Nathwani, A.C., Ng, C.Y., Zhou, J.F., Spence, Y., Nienhuis, A.W., Davidoff, A.M. (2003). Liver targeted delivery of recombinant adeno-associated virus (rAAV) serotype 5 vectors leads to sustained therapeutic expression of human factor IX (hFIX) in non-human primates previously sensitized to rAAV2 vectors..
Benjamin, R., Davidoff, A.M., Ng, C.Y.C., Pizzey, A., Yong, K., Nathwani, A.C. (2003). Murine models of hematological malignancies that mimic the disease phenotype in humans..
Porakishvili, N., Kulikova, N., Jewell, A.P., Youinou, P.Y., Yong, K., Nathwani, A., ...Hamblin, T.J. (2003). Preferential expression of CD180 by B cell chronic lymphocytic leukaemia (B-CLL) cells using IgVh mutated versus those using IgVh unmutated genes.
Davidoff, A.M., Ng, C.Y., Zhou, J., Spence, Y., Nathwani, A.C. (2003). Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway.. Blood, 102 (2), 480-488. doi:10.1182/blood-2002-09-2889

2002

Forbes, L.V., Gale, R.E., Pizzey, A., Pouwels, K., Nathwani, A., Linch, D.C. (2002). An activating mutation in the transmembrane domain of the granulocyte colony-stimulating factor receptor in patients with acute myeloid leukemia.. Oncogene, 21 (39), 5981-5989. doi:10.1038/sj.onc.1205767
Hanawa, H., Kelly, P.F., Nathwani, A.C., Persons, D.A., Vandergriff, J.A., Hargrove, P., ...Nienhuis, A.W. (2002). Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood.. Molecular Therapy, 5 (3), 242-251.
Davidoff, A.M., Nathwani, A.C., Spurbeck, W.W., Ng, C.Y., Zhou, J., Vanin, E.F. (2002). rAAV-mediated long-term liver-generated expression of an angiogenesis inhibitor can restrict renal tumor growth in mice.. Cancer Research, 62 (11), 3077-3083.
Nathwani, A., Davidoff, A.M., Hanawa, H., Hu, Y., Hoffer, F.A., Nikanorov, A., ...Lozier, J.N. (2002). Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques.. Blood, 100 (5), 1662-1669. doi:10.1182/blood-2002-02-0589

2001

Nathwani, A., Davidoff, A., Hanawa, H., Zhou, J.F., Vanin, E.F., Nienhuis, A.W. (2001). Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.. Blood, 97 (5), 1258-1265.
Kelly, P.F., Carrington, J., Nathwani, A., Vanin, E.F. (2001). RD114-pseudotyped oncoretroviral vectors. Biological and physical properties.. HEMATOPOIETIC STEM CELLS 2000 BASIC AND CLINICAL SCIENCES, 938 262-277.

2000

Nathwani, A.C., Hanawa, H., Vandergriff, J., Kelly, P., Vanin, E.F., Nienhuis, A.W. (2000). Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV.. Gene Therapy, 7 (3), 183-195.
Kelly, P.F., Vandergriff, J., Nathwani, A., Nienhuis, A.W., Vanin, E.F. (2000). Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein.. Blood, 96 (4), 1206-1214.

1999

Nathwani, A.C., Persons, D.A., Stevenson, S.C., Frare, P., McClelland, A., Nienhuis, A.W., Vanin, E.F. (1999). Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector.. Gene Therapy, 6 (8), 1456-1468.

1996

Durosinmi, M.A., Matutes, E., Ryan, K., Nathwani, A., Hedges, M., Catovsky, D. (1996). Royal Marsden teaching cases. An elderly man with weight loss and anaemia.. Leukemia and Lymphoma, 23 (1), 181-183.

1994

Nathwani, A.C., Gale, K.M., Pemberton, K.D., Crossman, D.C., Tuddenham, E.G., McVey, J.H. (1994). Efficient gene transfer into human umbilical vein endothelial cells allows functional analysis of the human tissue factor gene promoter.. British Journal of Haematology, 88 (1), 122-128.

1993

Nathwani, A.C., Goldman, J.M. (1993). Management of chronic myeloid leukaemia in lymphoid blast transformation.. Haematologica, 78 (3), 162-166.

1992

Nathwani, A.C., Tuddenham, E.G. (1992). Epidemiology of coagulation disorders.. Baillière's Clinical Haematology, 5 (2), 383-439.

1991

Vulliamy, T.J., Othman, A., Town, M., Nathwani, A., Falusi, Y., Luzzatto, L. (1991). Linkage disequilibrium of polymorphic sites in the G6PD gene in African populations and the origin of G6PD A-.. Gene Geography, 5 (1), 13-21.
Vulliamy, T.J., Othman, A., Town, M., Nathwani, A., Falusi, A.G., Mason, P.J., Luzzatto, L. (1991). Polymorphic sites in the African population detected by sequence analysis of the glucose-6-phosphate dehydrogenase gene outline the evolution of the variants A and A-.. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 88 (19), 8568-8571. doi:10.1073/pnas.88.19.8568

1990

Warren, A.J., Hegde, U.M., Nathwani, A., Reilly, I.A. (1990). Systemic vasculitis and myelodysplasia.. British Journal of Haematology, 75 (4), 627-629.

1900

Tuddenham, E.G.D., Nathwani, A.C., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D.C., ...Harrington, C. (1900). Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B.. N Engl J Med, 365
Nathwani, A.C., Cochrane, M., McIntosh, J., Ng, C.Y., Zhou, J., Gray, J.T., Davidoff, A.M. (1900). Enhancing transduction of the liver by adeno-associated viral vectors. Gene Therapy,
Caxaria, S.A. (1900). Induced pluripotent stem cells (iPSCs) for research and therapy: induction of hepatic differentiation in iPSCs and evaluation of their quality as a model of in vivo development in the context of coagulation. (Doctoral Dissertation). UCL (University College London).