Genomics of Personalized Medicine

Date/Time: Monday, October 5, 2020 - 10:00 AM – 12:00 PM
Track: Plenary Session
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Description:

Unraveling the genetic etiology of neurological disease provides fundamental insights into the cellular pathways underlying disease pathogenesis. It also facilitates disease modeling, as well as the design and testing of targeted therapeutics. This session will illustrate how genomics is paving the way for the field to move towards precision-based treatments. Pediatric neurologists have demonstrated that gene modification of a previously untreatable disease, spinal muscular atrophy, is feasible. Here, we will explore how genomic knowledge may be translated into disease-modifying interventions in other neurologic diseases.

Objective(s):

  • To review current genetic understanding of neurologic disease.

  • To delineate how genetic knowledge can be leveraged to advance the precision medicine paradigm.

Speaker(s):

CRISPR-Mediated Therapeutics for Duchenne Muscular Dystrophy


Genetic Discovery and Translation in Neuromuscular Diseases


Speaker(s):

Genomic Approaches Paving the Way for Precision Neurology


Patient-Customized Antisense Therapies for Rare Neurogenetic Diseases


Towards Primary Prevention of Genetic Prion Disease