Steven Gray, Ph.D.

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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.

• 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

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

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy.

Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ Mol Ther Methods Clin Dev 2018 Jun 9 160-171

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates.

Gray SJ, Matagne V, Bachaboina L, Yadav S, Ojeda SR, Samulski RJ Mol. Ther. 2011 Jun 19 6 1058-69

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.

Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M, Mol Ther 2022 May

Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome.

Ling Q, Rioux M, Hu Y, Lee M, Gray SJ, Mol Ther Methods Clin Dev 2021 Dec 23 158-168

Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice.

Judson MC, Shyng C, Simon JM, Davis CR, Punt AM, Salmon MT, Miller NW, Ritola KD, Elgersma Y, Amaral DG, Gray SJ, Philpot BD, JCI Insight 2021 Oct 6 20

Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice using an Intravenous Self-Complementary Hexosaminidase Vector.

Osmon KJ, Thompson P, Woodley E, Karumuthil-Melethil S, Heindel C, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS, Curr Gene Ther 2021 Sep

Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models.

Kot S, Karumuthil-Melethil S, Woodley E, Zaric V, Thompson P, Chen Z, Lykken E, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS, Int J Mol Sci 2021 Jun 22 13

Giant axonal neuropathy: cross sectional analysis of a large natural history cohort.

Bharucha-Goebel DX, Norato G, Saade D, Paredes E, Biancavilla V, Donkervoort S, Kaur R, Lehky T, Fink M, Armao D, Gray SJ, Waite M, Debs S, Averion G, Hu Y, Zein WM, Foley AR, Jain M, Bönnemann CG, Brain 2021 Jun

Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease.

Murray SJ, Russell KN, Melzer TR, Gray SJ, Heap SJ, Palmer DN, Mitchell NL, Exp Eye Res 2021 Jun 207 108600

 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.

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

• American Society of Gene & Cell Therapy (2015)
• 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)
• Hereditary Neuropathy Foundation Scientific Advisory Board (2014)
• Hunter’s Hope Foundation Scientific Advisory Board (2015)
• International Rett Syndrome Foundation Scientific Review Board (2011)
• Scientific Advisory Board, Opsin Therapeutics (2019)
• Scientific Advisory Board, Sarepta Therapeutics (2020)
• Scientific Advisory Board, Vertex Therapeutics (2019)
• Scientific Advisory Board, Vertex Therapeutics Lysogene (2019)