Company: Howard Hughes Medical Institute
Job title: Professor
Dr. Cepko trained in viral vector design as a postdoctoral fellow with Richard Mulligan, creating some of the first retroviral vectors. She now uses multiple types of viral vectors to address questions of cell fate determination and patterning in the vertebrate retina, as well as gene therapy for diseases that lead to blindness.
Exploring a Gene-Agnostic Therapeutic Approach to Target Multiple IRD Etiologies 11:50 am
There are too many disease genes (>200) leading to blindness to make single gene therapy realistic for a larger number of families There may be common problems across disease gene families that can be identified and approached using a more generic therapy We have developed an AAV gene therapy targeting inflammation, oxidative damage, and metabolic…Read more
day: Preclinical 3