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Dr. Steven Gray earned his Ph.D. in molecular biology from Vanderbilt University in 2006, after receiving a B.S. degree with honors from Auburn University. He performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at UNC Chapel Hill. He is currently an Associate Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center.

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 include preclinical studies for Rett Syndrome, Giant Axonal Neuropathy (GAN), Tay-Sachs, Krabbe, AGU, and Batten Disease, and have expanded into human clinical studies to test a gene therapy approach for GAN.

Dr. Gray has published over 50 peer-reviewed papers in journals such as New England Journal of Medicine, Molecular Therapy, Nature Biotechnology, Gene Therapy, and The Proceedings of the National Academy of Sciences. He also has 3 pending patents. His research is funded by the National Institute for Neurological Disorders and Stroke, as well as numerous large and small research foundations. Dr. Gray was recently recognized with 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


Featured Publications LegendFeatured Publications

Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery.
Sinnett SE, Hector RD, Gadalla KKE, Heindel C, Chen D, Zaric V, Bailey MES, Cobb SR, Gray SJ Mol Ther Methods Clin Dev 2017 Jun 5 106-115
Functional Analysis of the Ser149/Thr149 Variants of Human Aspartylglucosaminidase and Optimization of the Coding Sequence for Protein Production.
Banning A, König JF, Gray SJ, Tikkanen R Int J Mol Sci 2017 Mar 18 4
N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase.
Francis JS, Wojtas I, Markov V, Gray SJ, McCown TJ, Samulski RJ, Bilaniuk LT, Wang DJ, De Vivo DC, Janson CG, Leone P Neurobiol. Dis. 2016 Dec 96 323-334
Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria.
Banning A, Gülec C, Rouvinen J, Gray SJ, Tikkanen R Sci Rep 2016 Nov 6 37583
Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism.
Powell SK, Khan N, Parker CL, Samulski RJ, Matsushima G, Gray SJ, McCown TJ Gene Ther. 2016 Nov 23 11 807-814
Immunological considerations for treating globoid cell leukodystrophy.
Karumuthil-Melethil S, Gray SJ J. Neurosci. Res. 2016 11 94 11 1349-58
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 MS, Heindel C, Jakubauskas B, Bongarzone ER, Gray SJ J. Neurosci. Res. 2016 11 94 11 1138-51
Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease.
Armao D, Bailey RM, Bouldin TW, Kim Y, Gray SJ Clin. Auton. Res. 2016 Aug 26 4 307-13
Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.
Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, Mark BL, Mahuran D, Gray SJ, Walia JS Hum. Gene Ther. 2016 Jul 27 7 497-508
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.
Karumuthil-Melethil S, Nagabhushan Kalburgi S, Thompson P, Tropak M, Kaytor MD, Keimel JG, Mark BL, Mahuran D, Walia JS, Gray SJ Hum. Gene Ther. 2016 Jul 27 7 509-21


Featured Books Legend Featured Books

Honors & Awards

  • Outstanding New Investigator Award
    American Society of Gene and Cell Therapy (2019)
  • Health Care Hero Award
    Triangle Business Journal (2016)

Professional Associations/Affiliations

  • Scientific Advisory Board, Sarepta Therapeutics (2020)
  • Scientific Advisory Board, Opsin Therapeutics (2019)
  • Scientific Advisory Board, Vertex Therapeutics (2019)
  • Scientific Advisory Board, Vertex Therapeutics Lysogene (2019)
  • CMT Association STAR Advisory Board (2018)
  • Cure SPG47 Scientific Advisory Board (2016)
  • Foundation to Fight H-ABC Scientific Advisory Board (2016)
  • Galyatech, LLC Scientific Advisory Board (2016)
  • American Society of Gene & Cell Therapy (2015)
  • Hunter’s Hope Foundation Scientific Advisory Board (2015)
  • Hereditary Neuropathy Foundation Scientific Advisory Board (2014)
  • International Rett Syndrome Foundation Scientific Review Board (2011)