Steven Gray, Ph.D. Titles and Appointments Professor Schools Medical School | Graduate School Departments 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 Lafora disease gene therapy: EPM2A but not EPM2B overexpression results in Lafora body formation Alao EO, Sheibani M, Wu J, Marriam U, Evans D, Kasiri S, Verma M, Nitschke S, Nitschke F, Gray SJ, Mitra S, Minassian BA Neurotherapeutics 2026 Mar 23 RNAi-Induced Expression of Paternal UBE3A Kang HR, Zaric V, Rybalchenko V, Gray SJ, Butler RK Genes 2026 Feb 17 A Head-to-Head Comparison of AAV9 Biodistribution in Mice: Routes of Administration and Age Dependence Rioux M, Boitnott A, Paduri S, Hu Y, Gray SJ Genes 2026 Feb 17 Mechanisms of Adeno-Associated Virus Serotype 9 Vector Characterization and Quality Control through Solid-State Nanopores Thyashan N, Manawasinghe J, Gu C, Khatri S, Nelson C, Sanli ME, Gray SJ, Lee S, Dutta P, Alexandrakis G, Kim MJ ACS Nano 2026 Jan 20 2148-2163 First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial Greenberg BM, Minassian B, Messahel S, Edgar VB, Lowden A, Dahshi H, Nettesheim ER, Nguyen HH, Hughes S, Muthukumar AR, Srinivasan K, Iannaccone S, Varadarajan G, Gray SJ, Kayani SN EBioMedicine 2026 Jan 123 Should patients with pre-existing anti-AAV antibodies be excluded from receiving intrathecally delivered AAV vectors? Gray SJ Molecular Therapy Methods and Clinical Development 2025 Dec 33 Plasmonic Nanopore Sensing to Probe the DNA Loading Status of Adeno-Associated Viruses Renkes S, Gray SJ, Kim M, Alexandrakis G Chemosensors 2025 Dec 13 Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency Presa M, Bailey RM, Ray S, Bailey L, Tata S, Murphy T, Piec PA, Combs H, Gray SJ, Lutz C Communications Medicine 2025 Dec 5 T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN) Armao D, Bouldin TW, Bharucha-Goebel DX, Hartman TS, Gray SJ, Bailey RM, Saade D, Todd JJ, Jain M, Waite M, Bönnemann CG, Smith JK Muscle and Nerve 2025 Nov 72 1152-1155 Exploration of a DDX3X Gene Supplementation Therapy Including Expanded Characterization and Novel Findings of Sleep Disturbances in Ddx3x Haploinsufficient Mice Boitnott A, Jiji A, Plautz EJ, Hu Y, Chen X, Gray SJ Biological Psychiatry Global Open Science 2025 Nov 5 Results 1-10 of 119 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)
