Nathwani, Amit C.

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,864

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,273

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

Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 345

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 278

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

Davidoff, A. M., Ng, C. Y. C., Zhou, J., Spence, Y., & Nathwani, A. C. (2003) · Blood

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 244

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 161

Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial

Allay, J. A., Sleep, S., Long, S., Tillman, D. M., Clark, R., Carney, G., Fagone, P., McIntosh, J. H., Nienhuis, A. W., Davidoff, A. M., et al. (2011) · Human Gene Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 152

Gene Therapy for Hemophilia

Nathwani, A. C. (2019) · Hematology

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 140

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8

Natkunarajah, M., Trittibach, P., McIntosh, J., Duran, Y., Barker, S. E., Smith, A. J., Nathwani, A. C., & Ali, R. R. (2008) · Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 138

Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

Fagone, P., Wright, J. F., Nathwani, A. C., Nienhuis, A. W., Davidoff, A. M., & Gray, J. T. (2012) · Human Gene Therapy Methods

Virus-based gene therapy researchMolecular Biology Techniques and Applications

Cited by 97

Enhancing transduction of the liver by adeno-associated viral vectors

Nathwani, A. C., Cochrane, M., McIntosh, J., Ng, C. Y. C., Zhou, J., Gray, J. T., & Davidoff, A. M. (2009) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 77

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

McIntosh, J. H., Cochrane, M., Cobbold, S., Waldmann, H., Nathwani, S. A., Davidoff, A. M., & Nathwani, A. C. (2012) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 70

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

Nathwani, A. C., Tuddenham, E., Chowdary, P., McIntosh, J., Lee, D., Rosales, C., Phillips, M., Pie, J., Junfang, Z., Meagher, M. M., et al. (2018) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 52

Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps

Brimble, M. A., Cheng, P.-H., Winston, S. M., Reeves, I. L., Souquette, A., Spence, Y., Zhou, J., Wang, Y.-D., Morton, C. L., Valentine, M., et al. (2022) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 30

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

Osman, E., Evans, V., Graham, I. R., Athanasopoulos, T., McIntosh, J., Nathwani, A. C., Simons, J. P., Dickson, G., & Owen, J. S. (2009) · Atherosclerosis

Virus-based gene therapy researchCholesterol and Lipid Metabolism

Cited by 16

Our journey to successful gene therapy for hemophilia B

Nathwani, A. C., Nienhuis, A. W., & Davidoff, A. M. (2014) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 15

Mbd3 Promotes Reprogramming of Primary Human Fibroblasts

Jaffer, S., Goh, P., Abbasian, M., & Nathwani, A. C. (2018) · International Journal of Stem Cells

Pluripotent Stem Cells ResearchEpigenetics and DNA Methylation

Cited by 9