Byrne, Barry J.

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 954

Current clinical applications of in vivo gene therapy with AAVs

Mendell, J. R., Al-Zaidy, S. A., Rodino-Klapac, L. R., Goodspeed, K., Gray, S. J., Kay, C. N., Boye, S. L., Boye, S. E., George, L. A., Salabarria, S., et al. (2021) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 743

Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 406

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 327

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

Cideciyan, A. V., Hauswirth, W. W., Aleman, T. S., Kaushal, S., Schwartz, S. B., Boye, S. L., Windsor, E. A. M., Conlon, T. J., Sumaroka, A., Pang, J.-J., et al. (2009) · Human Gene Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 312

Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2D

Mendell, J. R., Rodino‐Klapac, L. R., Rosales, X. Q., Coley, B. D., Galloway, G., Lewis, S., Malik, V., Shilling, C., Byrne, B. J., Conlon, T., et al. (2010) · Annals of Neurology

Virus-based gene therapy researchMuscle Physiology and Disorders

Cited by 248

Gene Therapy for Aromatic l -Amino Acid Decarboxylase Deficiency

Hwu, W.-L., Muramatsu, S.-I., Tseng, S.-H., Tzen, K.-Y., Lee, N.-C., Chien, Y.-H., Snyder, R. O., Byrne, B. J., Tai, C.-H., & Wu, R.-M. (2012) · Science Translational Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 246

Acid a-Glucosidase Deficiency (Glycogenosis Type II, Pompe Disease)

Raben, N., Plotz, P., & Byrne, B. (2002) · Current Molecular Medicine

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 241

Structural insight into the unique properties of adeno-associated virus serotype 9

DiMattia, M. A., Nam, H.-J., Van Vliet, K., Mitchell, M., Bennett, A., Gurda, B. L., McKenna, R., Olson, N. H., Sinkovits, R. S., Potter, M., et al. (2012) · Journal of Virology

Virus-based gene therapy researchParvovirus B19 Infection Studies

Cited by 219

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

Salabarria, S. M., Corti, M., Coleman, K. E., Wichman, M. B., Berthy, J. A., D’Souza, P., Tifft, C. J., Herzog, R. W., Elder, M. E., Shoemaker, L. R., et al. (2024) · Journal of Clinical Investigation

Virus-based gene therapy researchComplement system in diseases

Cited by 159

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Smith, B. K., Collins, S. W., Conlon, T. J., Mah, C. S., Lawson, L. A., Martin, A. D., Fuller, D. D., Cleaver, B. D., Clément, N., Phillips, D., et al. (2013) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 158

Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial

Chien, Y.-H., Lee, N.-C., Tseng, S.-H., Tai, C.-H., Muramatsu, S.-I., Byrne, B. J., & Hwu, W.-L. (2017) · The Lancet Child & Adolescent Health

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 142

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

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV- hGAA ) Gene Therapy in Children Affected by Pompe Disease

Corti, M., Liberati, C., Smith, B. K., Lawson, L. A., Tuna, I. S., Conlon, T. J., Coleman, K. E., Islam, S., Herzog, R. W., Fuller, D. D., et al. (2017) · Human Gene Therapy Clinical Development

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 120

Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases

Perez, B. A., Shutterly, A., Chan, Y. K., Byrne, B. J., & Corti, M. (2020) · Brain Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 112

Structural studies of adeno-associated virus serotype 8 capsid transitions associated with endosomal trafficking

Nam, H.-J., Gurda, B. L., McKenna, R., Potter, M., Byrne, B., Salganik, M., Muzyczka, N., & Agbandje-McKenna, M. (2011) · Journal of Virology

Virus-based gene therapy researchParvovirus B19 Infection Studies

Cited by 108

Pompe disease gene therapy

Byrne, B. J., Falk, D. J., Pacak, C. A., Nayak, S., Herzog, R. W., Elder, M. E., Collins, S. W., Conlon, T. J., Clement, N., Cleaver, B. D., et al. (2011) · Human Molecular Genetics

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 91

A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study

Beggs, A. H., Byrne, B. J., De Chastonay, S., Haselkorn, T., Hughes, I., James, E. S., Kuntz, N. L., Simon, J., Swanson, L. C., Yang, M. L., et al. (2018) · Muscle & Nerve

Neurogenetic and Muscular Disorders ResearchMuscle Physiology and Disorders

Cited by 83

Tolerance Induction to Cytoplasmic β-Galactosidase by Hepatic AAV Gene Transfer — Implications for Antigen Presentation and Immunotoxicity

Martino, A. T., Nayak, S., Hoffman, B. E., Cooper, M., Liao, G., Markusic, D. M., Byrne, B. J., Terhorst, C., & Herzog, R. W. (2009) · PLoS ONE

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 54

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy

Byrne, B. J., Fuller, D. D., Smith, B. K., Clement, N., Coleman, K., Cleaver, B., Vaught, L., Falk, D. J., McCall, A., & Corti, M. (2019) · Annals of Translational Medicine

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 51

Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease

Todd, A. G., McElroy, J. A., Grange, R. W., Fuller, D. D., Walter, G. A., Byrne, B. J., & Falk, D. J. (2015) · Annals of Neurology

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 51

Innate immune sensing of adeno-associated virus vectors

Cao, D., Byrne, B. J., de Jong, Y. P., Terhorst, C., Duan, D., Herzog, R. W., & Kumar, S. R. P. (2024) · Human Gene Therapy

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 50

Atomic structure of a rationally engineered gene delivery vector, AAV2. 5

Burg, M., Rosebrough, C., Drouin, L. M., Bennett, A., Mietzsch, M., Chipman, P., McKenna, R., Sousa, D., Potter, M., Byrne, B., et al. (2018) · Journal of Structural Biology

Virus-based gene therapy researchEnzyme Structure and Function

Cited by 30

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver

Rana, J., Herzog, R. W., Muñoz-Melero, M., Yamada, K., Kumar, S. R. P., Lam, A. K., Markusic, D. M., Duan, D., Terhorst, C., Byrne, B. J., et al. (2024) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

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