Gray, Steven J.

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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine

Muscle Physiology and DisordersVirus-based gene therapy research

Cited by 655

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy

Virus-based gene therapy researchBarrier Structure and Function Studies

Cited by 501

Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors

Gray, S. J., Foti, S. B., Schwartz, J. W., Bachaboina, L., Taylor-Blake, B., Coleman, J., Ehlers, M. D., Zylka, M. J., McCown, T. J., & Samulski, R. J. (2011) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 420

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 400

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gray, S. J., Nagabhushan Kalburgi, S., McCown, T. J., & Jude Samulski, R. (2013) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 377

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy

Virus-based gene therapy researchBarrier Structure and Function Studies

Cited by 185

Rett Syndrome: Crossing the Threshold to Clinical Translation

Katz, D. M., Bird, A., Coenraads, M., Gray, S. J., Menon, D. U., Philpot, B. D., & Tarquinio, D. C. (2016) · Trends in Neurosciences

Genetics and Neurodevelopmental DisordersAutism Spectrum Disorder Research

Cited by 150

Methods for Gene Transfer to the Central Nervous System

Kantor, B., Bailey, R. M., Wimberly, K., Kalburgi, S. N., & Gray, S. J. (2014) · Advances in Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 109

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 93

Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

Bailey, R. M., Rozenberg, A., & Gray, S. J. (2020) · Brain Research

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 83

Clinical Applications Involving CNS Gene Transfer

Kantor, B., McCown, T., Leone, P., & Gray, S. J. (2014) · Advances in Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 74

Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates

Mandel, R. J., Marmion, D. J., Kirik, D., Chu, Y., Heindel, C., McCown, T., Gray, S. J., & Kordower, J. H. (2017) · Acta Neuropathologica Communications

Parkinson's Disease Mechanisms and TreatmentsVirus-based gene therapy research

Cited by 56

Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant

Karumuthil‐Melethil, S., Marshall, M. S., Heindel, C., Jakubauskas, B., Bongarzone, E. R., & Gray, S. J. (2016) · Journal of Neuroscience Research

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 51

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