Telegram: Rare disease research at UMMS holds deep meaning for families
|Rachel Epstein, center, visits Guangping Gao, PhD, and intern Jessica Epstein, her sister, at Dr. Gao’s lab in 2014.|
Guangping Gao, PhD, had just arrived at UMass Medical School to lead a new gene therapy research center, about 10 years ago, when he was contacted by UMMS employee Sherry Epstein about her daughter Rachel’s rare disease, called Canavan. Dr. Gao, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, and director of the Horae Gene Therapy Center & Vector Core, had studied extensively the debilitating neurological disease that deprives patients of the ability to walk, sit or talk.
Soon, Gao met Rachel. “That moment when I saw her, I know that is my major focus of my career at UMass,” Gao told the Telegram & Gazette for a March 4 article.
Now Gao is nearing clinical trials for a gene therapy treatment for Canavan. He is co-director of the new Li Weibo Institute for Rare Diseases Research. Sherri Epstein credits Gao and his colleagues at UMMS with making a difference in the lives of families with rare diseases, such as hers.
Learn more in the full Telegram story, below.
Related links on UMassMedNow:
Synergy among 40 scientists at Li Weibo Institute for Rare Diseases Research accelerates discovery
Newsweek: UMass Medical School develops gene therapy for rare neurodegenerative disease