Steven J. Gray
Affiliations: | 2008-2018 | Gene Therapy Center | University of North Carolina, Chapel Hill, Chapel Hill, NC |
| 2017- | Neurology and Pediatrics | University of Texas Southwestern Medical Center, Dallas, TX, United States |
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Cross-listing: Cell & Gene Therapy Tree
Parents
Sign in to add mentor| Ellen Fanning | grad student | 2000-2006 | Vanderbilt (Chemistry Tree) |
| Jude Samulski | post-doc | 2006-2008 | UNC Chapel Hill (Cell & Gene Therapy Tree) |
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Publications
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| Ryckman AE, Deschenes NM, Quinville BM, et al. (2023) Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study. Molecular Therapy. Methods & Clinical Development. 32: 101168 |
| Gumusgoz E, Kasiri S, Verma M, et al. (2023) CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy. Gene Therapy |
| Murray SJ, Wellby MP, Barrell GK, et al. (2023) Efficacy of dual intracerebroventricular and intravitreal gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease. Frontiers in Pharmacology. 14: 1212235 |
| Vyas M, Deschenes NM, Osmon KJL, et al. (2023) Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. International Journal of Molecular Sciences. 24 |
| Ling Q, Herstine JA, Bradbury A, et al. (2023) AAV-based in vivo gene therapy for neurological disorders. Nature Reviews. Drug Discovery |
| Casy W, Garza IT, Chen X, et al. (2023) SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution. Genes. 14 |
| Mitchell NL, Murray SJ, Wellby MP, et al. (2023) Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease. Frontiers in Genetics. 14: 1212228 |
| Wong H, Hooper AW, Kang HR, et al. (2023) CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice. Jci Insight. 8 |
| Chen X, Lim DA, Lawlor MW, et al. (2023) Biodistribution of adeno-associated virus gene therapy following CSF-directed administration. Human Gene Therapy |
| Chen X, Dong T, Hu Y, et al. (2022) AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease. The Journal of Clinical Investigation |