Gene Therapy 101

The July CRPTS session will be given by Tabatha Simmons, Manager of Gene Therapy-Research Unit at Nationwide Children's Hospital.

This presentation will include an introduction to the biology of gene therapy, historical highlights over the last 20 years and what it looks like today-both in the clinical research and in the clinical setting. We will further discuss the ethical implications of on-time treatments and what these therapies mean for the rare disease communities.