Most rare diseases are caused by genetic mutations. Often, the mutations result in a protein that does not have the shape necessary to successfully perform its function. New approaches are being developed to address these causes of rare disease through cell therapies or pharmacological chaperones which help proteins take on the shape needed to get to where they need to be within cells. Modified cells, or cells combined with other molecules, may also correct the genetic problem in tissues, or replace tissues entirely. Join us for a discussion of these new approaches and how they may be used as rare disease therapies.
Speaker: Shaun Lee, PhD, Monahan Family Associate Professor of Rare and Neglected Diseases, University of Notre Dame
Speaker: Weston Miller, MD, Medical Director, Sangamo Therapeutics
Day 2, Track 4