Nienhuis, Arthur W.

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

5-Azacytidine Selectively Increases γ-Globin Synthesis in a Patient with β+Thalassemia

Ley, T. J., DeSimone, J., Anagnou, N. P., Keller, G. H., Humphries, R. K., Turner, P. H., Young, N. S., Heller, P., & Nienhuis, A. W. (1982) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersHemoglobin structure and function

Cited by 490

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

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

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

Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells

Leimig, T., Mann, L., Martin, M. D. P., Bonten, E., Persons, D., Knowles, J., Allay, J. A., Cunningham, J., Nienhuis, A. W., Smeyne, R., et al. (2002) · Blood

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 75

Development of Gene Therapy for Thalassemia

Nienhuis, A. W., & Persons, D. A. (2012) · Cold Spring Harbor Perspectives in Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 38

Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis

Hu, H., Gomero, E., Bonten, E., Gray, J. T., Allay, J., Wu, Y., Wu, J., Calabrese, C., Nienhuis, A., & d'Azzo, A. (2012) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 37

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

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector

Singh, S., Khan, I., Khim, S., Seymour, B., Sommer, K., Wielgosz, M., Norgaard, Z., Kiem, H.-P., Adair, J., Liggitt, D., et al. (2017) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 13