Steven Gray, Ph.D. Titles and Appointments Professor School Medical School Department Pediatrics | Eugene McDermott Center for Human Growth and Development | Molecular Biology | Neurology Graduate Programs Genetics, Development and Disease Biography Download Curriculum Vitae Dr. Steven Gray received a B.S. degree with honors from Auburn University followed by a Ph.D. in molecular biology from Vanderbilt University and postdoctoral training at the UNC Chapel Hill Gene Therapy Center. He is currently a Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center. Dr. Gray is the co-director of the UTSW Gene Therapy Program and director of the UTSW Viral Vector Facility. He maintains affiliations with the Department of Molecular Biology, the Department of Neurology, and the Eugene McDermott Center for Human Growth and Development at UT Southwestern. Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system. His major focus is in AAV vector development to develop vectors tailored to serve specific clinical and research applications involving the nervous system. These include the development of novel AAV capsids amenable to widespread CNS gene transfer. As AAV-based platform gene transfer technologies have been developed to achieve global, efficient, and in some cases cell-type specific CNS gene delivery, his research focus has also included preclinical studies to apply these reagents toward the development of treatments for neurological diseases. Currently these have resulted in approved and ongoing human clinical trials to test gene therapies for the following disorders: Rett Syndrome, Giant Axonal Neuropathy (GAN), Tay-Sachs, Sandhoff, Spastic Paraplegia Type 50, and Batten Diseases (CLN1, CLN5, and CLN7). Dr. Gray has published over 90 peer-reviewed papers in journals such as New England Journal of Medicine, Molecular Therapy, Brain, Journal of Clinical Investigations, Nature Biotechnology, Nature Medicine, and The Proceedings of the National Academy of Sciences. He also has over 20 pending or awarded patents. His research is funded by the National Institute for Neurological Disorders and Stroke, as well as numerous large and small research foundations and industry partners. Dr. Gray was recognized with the American Society of Gene and Cell Therapy’s Outstanding Young Investigator Award in 2019, the 2016 Healthcare Hero award by the Triangle Business Journal, and his work on GAN was featured in a story by the CBS National Evening News in 2015. Research Interest Adeno-Associated Virus (AAV) vector engineering Developing AAV vector-based treatments (gene therapy) for nervous system diseases Facilitating bench-to-bedside translation of gene therapy into Phase I clinical trials Improving AAV vector manufacturing processes Understanding and overcoming immune responses associated with viral vector-based gene therapy approaches Publications Featured Publications AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient. Dowling JJ, Pirovolakis T, Devakandan K, Stosic A, Pidsadny M, Nigro E, Sahin M, Ebrahimi-Fakhari D, Messahel S, Varadarajan G, Greenberg BM, Chen X, Minassian BA, Cohn R, Bonnemann CG, Gray SJ, Nat Med 2024 Jun Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies. Chen X, Dong T, Hu Y, De Pace R, Mattera R, Eberhardt K, Ziegler M, Pirovolakis T, Sahin M, Bonifacino JS, Ebrahimi-Fakhari D, Gray SJ, J Clin Invest 2023 May 133 10 AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease. Chen X, Dong T, Hu Y, Shaffo FC, Belur NR, Mazzulli JR, Gray SJ, J Clin Invest 2022 Jan Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study. Ryckman AE, Deschenes NM, Quinville BM, Osmon KJL, Mitchell M, Chen Z, Gray SJ, Walia JS, Mol Ther Methods Clin Dev 2024 Mar 32 1 101168 Intrathecal Gene Therapy for Giant Axonal Neuropathy. Bharucha-Goebel DX, Todd JJ, Saade D, Norato G, Jain M, Lehky T, Bailey RM, Chichester JA, Calcedo R, Armao D, Foley AR, Mohassel P, Tesfaye E, Carlin BP, Seremula B, Waite M, Zein WM, Huryn LA, Crawford TO, Sumner CJ, Hoke A, Heiss JD, Charnas L, Hooper JE, Bouldin TW, Kang EM, Rybin D, Gray SJ, Bönnemann CG, N Engl J Med 2024 Mar 390 12 1092-1104 CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy. Gumusgoz E, Kasiri S, Verma M, Wu J, Villarreal Acha D, Marriam U, Fyffe-Maricich S, Lin A, Chen X, Gray SJ, Minassian BA, Gene Ther 2023 Dec The Efficacy of a Human-Ready miniMECP2 Gene Therapy in a Pre-Clinical Model of Rett Syndrome. Sadhu C, Lyons C, Oh J, Jagadeeswaran I, Gray SJ, Sinnett SE, Genes (Basel) 2023 Dec 15 1 AAV-based in vivo gene therapy for neurological disorders. Ling Q, Herstine JA, Bradbury A, Gray SJ, Nat Rev Drug Discov 2023 Oct 22 10 789-806 Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Vyas M, Deschenes NM, Osmon KJL, Chen Z, Ahmad I, Kot S, Thompson P, Richmond C, Gray SJ, Walia JS, Int J Mol Sci 2023 Sep 24 19 SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution. Casy W, Garza IT, Chen X, Dong T, Hu Y, Kanchwala M, Trygg CB, Shyng C, Xing C, Bunnell BA, Braun SE, Gray SJ, Genes (Basel) 2023 Aug 14 8 Results 1-10 of 88 1 2 3 4 5 Next Last Books Featured Books Methods for Gene Transfer to the Central Nervous System (Kantor B, Bailey RM, Wimberly K, Kalburgi SN, and Gray SJ). In Advances in Genetics, Volume 87 T. Friedmann, J.C. Dunlap, & S.F. Goodwin (Ed.) (2014). London, Academic Press Clinical Applications Involving CNS Gene Transfer (Kantor B, McCown T, Leone P, and Gray SJ). In Advances in Genetics, Volume 87 T. Friedmann, J.C. Dunlap, & S.F. Goodwin (Ed.) (2014). London, Academic Press Chapter contribution to Structural Virology (Dismuke D, Gray SJ, Hirsch M, Samulski RJ, and Muzyczka N). In Structural Virology Agbandje-McKenna and McKenna (Ed.) (2011). London, RSC Publishing Production of Recombinant Adeno-Associated Viral Vectors and Use in In Vitro and In Vivo Administration (Chapter 4: Unit 4.17). In Current Protocols in Neuroscience Gray SJ, Choi VW, Asokan A, Haberman RA, McCown TJ, and Samulski RJ (2011). John Wiley & Sons, Inc. Honors & Awards Thomas Dierks AwardRemarkable contributions to Multiple Sulfatase Deficiency Research (2022) Outstanding New Investigator AwardAmerican Society of Gene and Cell Therapy (2019) Health Care Hero AwardTriangle Business Journal (2016) Professional Associations/Affiliations Abstract Review Chair, American Society for Gene and Cell Therapy, Neurologic Disease Category (2024) Associate Editor, Neurosurgery (2014-2017) Board of Directors, Elpida Therapeutics (2023) Chief Scientific Advisor, Taysha Gene Therapies (2020) Committee Member, Neurologic & Ophthalmic Gene & Cell Therapy, American Society for Cell and Gene Therapy (2014-2020) Scientific Advisory Board, CDKL5 Research Collaborative (2017-2022) Scientific Advisory Board, CMT Association STAR Advisory Board (2018) Scientific Advisory Board, Codexis (2021-2023) Scientific Advisory Board, Cure SPG47 (2016-2022) Scientific Advisory Board, Forge Biologics (2021) Scientific Advisory Board, Form Bio (2023) Scientific Advisory Board, Foundation to Fight H-ABC (2016-2022) Scientific Advisory Board, FOXG1 Foundation (2017-2022) Scientific Advisory Board, Galyatech, LLC (2016-2022) Scientific Advisory Board, Hereditary Neuropathy Foundation (2014-2020) Scientific Advisory Board, Hunter's Hope Foundation (2015-2022) Scientific Advisory Board, Lysogene (2019-2021) Scientific Advisory Board, Nanoscope Therapeutics (2021) Scientific Advisory Board, Opsin Therapeutics (2019) Scientific Advisory Board, Sarepta Therapeutics (2019) Scientific Advisory Board, Vertex Therapeutics (2019-2021) Scientific Review Board, International Rett Syndrome Foundation (2011)
