Over the past decade, neuroscientists have made tremendous breakthroughs in understanding the underlying causes of brain disease. At the same time, CRISPR genome editing has generated new hope for developing treatments for a wide range of unaddressed genetic diseases. Now a group of leading researchers at UC Berkeley, UCSF, and the Innovative Genomics Institute, are bringing these two advancements together to create new, breakthrough therapies for neurological diseases.
In this roundtable discussion, moderated by IGI Public Impact Director Melinda Kliegman, the panelists discuss the current state of neurological disease research (3:03–9:01), what this new partnership will open up (9:04–14:50), the role of CRISPR-Cas9 in this effort (14:49–17:13), current challenges, including delivery of genome-editing therapies and high degrees of failure of clinical trials (17:15–24:20), translating fundamental research to applicable solutions (24:22–29:40), the potential of new CRISPR-based technology for treating neurological diseases (29:42–31:30), when these new therapies could be available (31:32–38:28), equitable access to new therapies (38:29–42:38), and more (42:41–end).
Read more about the recently announced Alliance for Therapies in Neuroscience (ATN), a long-term research partnership with Genentech and its parent company, Roche Holding AG, to speed the development of new therapeutics for debilitating brain diseases and disorders of the central nervous system.
Jennifer Doudna — Jennifer A. Doudna, Ph.D., is the Li Ka Shing Chancellor’s Chair and a Professor in the Departments of Chemistry and of Molecular and Cell Biology at UC Berkeley and the Founder and President of the Innovative Genomics Institute. Her groundbreaking development of CRISPR-Cas9 as a genome-engineering technology, with collaborator Emmanuelle Charpentier, earned the two the 2020 Nobel Prize in Chemistry and forever changed the course of human and agricultural genomics research.
Stephen Hauser — Stephen L. Hauser, M.D., is the Robert A. Fishman Distinguished Professor of Neurology at the University of California, San Francisco and Director of Weill Neurohub West. He is also Director of the UCSF Weill Institute for Neurosciences, an umbrella organization that links the clinical and basic neurosciences at UCSF to accelerate research against neurologic diseases. A neuroimmunologist, Dr. Hauser’s research has advanced our understanding of the genetic basis, immune mechanisms, and treatment of multiple sclerosis (MS).
Ehud Isacoff —Ehud Y. Isacoff, Ph.D., is Evan Rauch Professor of Neuroscience and Director of both the Helen Wills Neuroscience Institute and of Weill Neurohub East at UC Berkeley. He is a biophysicist and neuroscientist, and a pioneer in the field of optogenetics research, using light to study neural circuits and neural transmission. He applies this research to neuronal diseases, including retinal disease, to restore neural function.