GRTC Lecture Series
Utilizing and expanding AAV platform approaches to treat neurological disorders
Dear colleagues,
It is our pleasure to invite you to the upcoming talk in the GRTC Lecture Series, featuring Dr. Steven Gray from the Eugene McDermott Center for Human Growth and Development, UT Southwestern (Texas, USA).
🗓 Date: 09.03.2026
🕒 Time: 4-5pm (CET)
🔗 Zoom Link: https://med-uni-tuebingen-de.zoom-x.de/j/66960639584?pwd=9hWXZJ92FBS26PXavhahdGQZbzKSok.1
🔑 Meeting ID: 669 6063 9584
🔒 Passcode: 243074
Title: Utilizing and expanding AAV platform approaches to treat neurological disorders
Abstract: Nervous system-directed gene therapy has expanded substantially over the last decade following regulatory approval of IV-administered and AAV9-based Zolgensma as a treatment for Spinal Muscular Atrophy. The possibility of treating many neurological disorders with the same general approach has been extremely appealing, but many practical and safety concerns shifted to utilizing AAV9 vectors injected into the cerebrospinal fluid (CSF) to broadly direct gene therapy to the brain. The number of clinical trial utilizing intra-CSF delivery of AAV9 has expanded rapidly, and this emerged as a platform-type of approach. The advantages of using this platform approach are numerous, even with inherent limitations associated with AAV9. However, recent profound advancements in engineered AAV technology are poised to greatly expand the potential for nervous-system directed gene therapy. Is this the beginning of a new platform?
Speaker Bio: Dr. Steven Gray received a B.S. degree with honors from Auburn University followed by a Ph.D. in molecular biology from Vanderbilt University and postdoctoral training at the UNC Chapel Hill Gene Therapy Center. He is currently a Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center. Dr. Gray is the co-director of the UTSW Gene Therapy Program and director of the UTSW Viral Vector Facility. He maintains affiliations with the Department of Molecular Biology, the Department of Neurology, and the Eugene McDermott Center for Human Growth and Development 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 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 have resulted in approved and ongoing human clinical trials to test gene therapies for the following disorders: Rett Syndrome, Giant Axonal Neuropathy (GAN), Tay-Sachs, Sandhoff, Spastic Paraplegia Type 50, and Batten Diseases (CLN1, CLN5, and CLN7). (https://profiles.utsouthwestern.edu/profile/175486/steven-gray.html)
Please feel free to share this invitation with colleagues or others who may be interested in attending.
We are looking forward to your participation!
