Steven Gray, Ph.D.
Associate Professor

Download Curriculum Vitae

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

Optical coherence tomography features in brothers with aspartylglucosaminuria.

Goodspeed K, Harder L, Hughes S, Conger D, Taravella J, Gray SJ, Minassian B Ann Clin Transl Neurol 2018 Dec 5 12 1622-1626

Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.

Mitchell NL, Russell KN, Wellby MP, Wicky HE, Schoderboeck L, Barrell GK, Melzer TR, Gray SJ, Hughes SM, Palmer DN Mol. Ther. 2018 Jul

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

Recent progress and considerations for AAV gene therapies targeting the central nervous system.

Lykken EA, Shyng C, Edwards RJ, Rozenberg A, Gray SJ J Neurodev Disord 2018 May 10 1 16

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.

Marshall MS, Issa Y, Jakubauskas B, Stoskute M, Elackattu V, Marshall JN, Bogue W, Nguyen D, Hauck Z, Rue E, Karumuthil-Melethil S, Zaric V, Bosland M, van Breemen RB, Givogri MI, Gray SJ, Crocker SJ, Bongarzone ER Mol. Ther. 2018 Jan

Correction: Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation.

Ramsingh AI, Gray SJ, Reilly A, Koday M, Bratt D, Koday MT, Munson P, Murnane R, Smedley J, Hu Y, Messer A, Fuller DH PLoS ONE 2018 13 11 e0207077

Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation.

Ramsingh AI, Gray SJ, Reilly A, Koday M, Bratt D, Koday MT, Murnane R, Smedley J, Hu Y, Messer A, Fuller DH PLoS ONE 2018 13 6 e0198154

Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome.

Sinnett SE, Gray SJ Discov Med 2017 Oct 24 132 153-159

Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates.

Mandel RJ, Marmion DJ, Kirik D, Chu Y, Heindel C, McCown T, Gray SJ, Kordower JH Acta Neuropathol Commun 2017 Jun 5 1 47

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome.

Gadalla KKE, Vudhironarit T, Hector RD, Sinnett S, Bahey NG, Bailey MES, Gray SJ, Cobb SR Mol Ther Methods Clin Dev 2017 Jun 5 180-190

 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)

• 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)