Monday, November 15 | 2:00 pm - 2:30 pm

Reaching the Brain: Developing Novel Genome Engineering Delivery Systems in vivo

Kyle Fink, PhD

Assistant Professor
Neurology Department & Stem Cell Program
Institute for Regenerative Cures
University of California, Davis Medical Center

Abstract

Recent breakthroughs in the use of CRISPR/Cas9 and other genetic modifying domains have revolutionized the way we develop treatments for rare neurological disorders. However, prior to applying these breakthroughs clinically, several major hurdles exist that must be addressed in pre-clinical studies. For many neurodevelopmental disorders and neurodegenerative diseases, accessing the affected cells in the brain remains a significant obstacle. The Fink lab has undertaken multiple approaches to address this problem. Specifically, we developed a novel AAV vector system to deliver CRISPR/dCas9 epigenetic editors to the brain as well as stem cell-mediated delivery platforms to secrete DNA-binding domains such as Zinc Fingers, for regulating gene expression in vivo. To assess global distribution and efficacy across the mouse brain, we utilize the ZEISS AxioScan high throughput slide scanner; enabling screening and consequently research progression to happen much quicker than previously possible. Brains of particular interest are then examined in 3D at high resolution for colocalization and subcellular expression using the Apotome on a ZEISS Axio Observer. Taken together these studies and approaches are helping to move these promising interventions down the translational pipeline toward treatment.

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