CRISPR gene editing and stem cells show promise as potential therapies anticipated to have a major impact on treating rare diseases. These technologies are already making an impact as research tools used to uncover the underlying mechanisms of rare diseases and the testing of other potential therapies. CRISPR and induced pluripotent stem (iPS) cells can reduce the time and effort to conduct research on rare diseases by making it easier to develop animal models or by supplementing animal model research. This session will provide an overview of these approaches, focusing on the benefits of including CRISPR and iPS cell approaches in a rare disease research portfolio.
Speaker: Marcelo Coba, PhD, Assistant Professor of Psychiatry, Zilkha Neurogenetic Institute, Keck School of Medicine of University of Southern California
Speaker: Lisa Flint, Founder and Managing Director, The AADC Research Trust
Day 1, Track 4