Samulski, R. Jude

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

Wu, Z., Sun, J., Zhang, T., Yin, C., Yin, F., Van Dyke, T., Samulski, R. J., & Monahan, P. E. (2008) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 153

Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’

Kishimoto, T. K., & Samulski, R. J. (2022) · Expert Opinion on Biological Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 152

Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing

Choi, V. W., McCarty, D. M., & Samulski, R. J. (2006) · Journal of Virology

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 144

Novel Transcriptional Regulatory Signals in the Adeno-Associated Virus Terminal Repeat A/D Junction Element

Haberman, R. P., McCown, T. J., & Samulski, R. J. (2000) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 139

Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus

Rabinowitz, J. E., Xiao, W., & Samulski, R. J. (1999) · Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 136

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., et al. (2010) · Human Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 131

Capsid antibodies to different adeno-associated virus serotypes bind common regions

Gurda, B. L., DiMattia, M. A., Miller, E. B., Bennett, A., McKenna, R., Weichert, W. S., Nelson, C. D., Chen, W.-J., Muzyczka, N., Olson, N. H., et al. (2013) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 127

Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application

Monahan, P. E., Lothrop, C. D., Sun, J., Hirsch, M. L., Kafri, T., Kantor, B., Sarkar, R., Tillson, D. M., Elia, J. R., & Samulski, R. J. (2010) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 125

Adeno-Associated Virus (AAV) Versus Immune Response

Rabinowitz, J., Chan, Y. K., & Samulski, R. J. (2019) · Viruses

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 122

Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction

Shao, W., Earley, L. F., Chai, Z., Chen, X., Sun, J., He, T., Deng, M., Hirsch, M. L., Ting, J., Samulski, R. J., et al. (2018) · JCI Insight

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 119

Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression

Earley, L. F., Conatser, L. M., Lue, V. M., Dobbins, A. L., Li, C., Hirsch, M. L., & Samulski, R. J. (2020) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 111

Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism

Powell, S. K., Khan, N., Parker, C. L., Samulski, R. J., Matsushima, G., Gray, S. J., & McCown, T. J. (2016) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 92

Surface-exposed adeno-associated virus Vp1-NLS capsid fusion protein rescues infectivity of noninfectious wild-type Vp2/Vp3 and Vp3-only capsids but not that of fivefold pore mutant virions

Grieger, J. C., Johnson, J. S., Gurda-Whitaker, B., Agbandje-McKenna, M., & Samulski, R. J. (2007) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 83

Cellular immune response to cryptic epitopes during therapeutic gene transfer

Li, C., Goudy, K., Hirsch, M., Asokan, A., Fan, Y., Alexander, J., Sun, J., Monahan, P., Seiber, D., Sidney, J., et al. (2009) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 79

Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration

Zhang, X., He, T., Chai, Z., Samulski, R. J., & Li, C. (2018) · Biomaterials

Barrier Structure and Function StudiesVirus-based gene therapy research

Cited by 65

Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors

Mitchell, A. M., & Samulski, R. J. (2013) · Journal of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 63

Oversized AAV Transductifon Is Mediated via a DNA-PKcs-independent, Rad51C-dependent Repair Pathway

Hirsch, M. L., Li, C., Bellon, I., Yin, C., Chavala, S., Pryadkina, M., Richard, I., & Samulski, R. J. (2013) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 62

Adeno-associated virus capsid antigen presentation is dependent on endosomal escape

Li, C., He, Y., Nicolson, S., Hirsch, M., Weinberg, M. S., Zhang, P., Kafri, T., & Samulski, R. J. (2013) · Journal of Clinical Investigation

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 61

Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism

Woodard, K. T., Liang, K. J., Bennett, W. C., & Samulski, R. J. (2016) · Journal of Virology

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 59

Ocular gene transfer with self-complementary AAV vectors

Yokoi, K., Kachi, S., Zhang, H. S., Gregory, P. D., Spratt, S. K., Samulski, R. J., & Campochiaro, P. A. (2007) · Investigative Opthalmology & Visual Science

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 58

Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells

Hirsch, M. L., Fagan, B. M., Dumitru, R., Bower, J. J., Yadav, S., Porteus, M. H., Pevny, L. H., & Samulski, R. J. (2011) · PLoS ONE

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 53

Production of recombinant adeno-associated viral vectors for in vitro and in vivo use

Choi, V. W., Asokan, A., Haberman, R. A., & Samulski, R. J. (2007) · Current Protocols in Molecular Biology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 52

N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase

Francis, J. S., Wojtas, I., Markov, V., Gray, S. J., McCown, T. J., Samulski, R. J., Bilaniuk, L. T., Wang, D.-J., De Vivo, D. C., Janson, C. G., et al. (2016) · Neurobiology of Disease

Metabolism and Genetic DisordersVirus-based gene therapy research

Cited by 49

Insight into the mechanism of inhibition of adeno-associated virus by the Mre11/Rad50/Nbs1 complex

Lentz, T. B., & Samulski, R. J. (2015) · Journal of Virology

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 48