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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

Insights into the Pathogenesis and Treatment of Krabbe Disease.
Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS Pediatr Endocrinol Rev 2016 Jun 13 Suppl 1 689-96
Timing of Gene Therapy Interventions: The Earlier, the Better.
Gray SJ Mol. Ther. 2016 06 24 6 1017-1018
Rett Syndrome: Crossing the Threshold to Clinical Translation.
Katz DM, Bird A, Coenraads M, Gray SJ, Menon DU, Philpot BD, Tarquinio DC Trends Neurosci. 2016 Feb 39 2 100-13
Construction of a hybrid ß-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo.
Tropak MB, Yonekawa S, Karumuthil-Melethil S, Thompson P, Wakarchuk W, Gray SJ, Walia JS, Mark BL, Mahuran D Mol Ther Methods Clin Dev 2016 3 15057


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)