Gene therapy in Paediatric CNS Disorders

Meet the speaker

Steven Gray, Ph.D
Associate Professor
Department of Pediatrics
University of Texas Southwestern Medical Center
Email: steven.gray@utsouthwestern.edu

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 is the director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development, and the Hamon Center for Regenerative Science and Medicine 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 research focus has also included preclinical studies to development AAV-based treatments for neurological diseases, some of which have translated into clinical trials.

About the talk

Gene therapy is an emerging area of medicine, which is now showing profound therapeutic benefit for disorders such as spinal muscular atrophy. Vectors based on adeno-associated virus (AAV), particularly AAV9, are capable of broad CNS gene transfer and can be viewed as a platform approach to treat a variety of inherited CNS disorders. Dr. Gray will present the current state-of-the-art on AAV-mediated CNS gene transfer, and provide multiple examples of small and large animal models that have been successfully treated with AAV9 vectors including Giant Axonal Neuropathy, Krabbe diseases, CLN1 Batten disease, CLN7 Batten disease, CLN5 Batten disease, and Aspartylglucosaminuria.

Learning objectives

Attendees will

• learn about the current state-of-the-art methods for gene transfer to the central nervous system using AAV vectors
• see examples of disorders where current gene therapy approaches can confer substantial treatment benefits, compared to other disorders where treatment effects may be more modest
• gain some understanding of the promise as well as limitations of current AAV gene therapy capabilities.
• gain an appreciation of this emerging area of medicine to potentially bring treatments to hundreds or single-gene rare diseases.