|Michael Green, MD, PhD|
|Guangping Gao, PhD|
Inspired by patients and their families, 40 scientists at the The Li Weibo (李伟波) Institute for Rare Diseases Research at UMass Medical School are working together to discover cures for dozens of rare disorders, according to co-directors Michael Green, MD, PhD, and Guangping Gao, PhD.
“We think that even though there are 7,000 different rare diseases, (as identified by the National Institutes of Health), there are far fewer than 7,000 different causes,” said Dr. Green, Howard Hughes Medical Institute investigator, the Lambi and Sarah Adams Chair of Genetic Research, and chair and professor of molecular, cell & cancer biology, in a Feb. 27 Facebook Live broadcast on the importance of Rare Disease Day. “By putting these people together, we are drawing on each other’s common experience and expertise. I think we will be able to have effects on more than one of the rare diseases.”
The institute was launched last fall, supported by a $10 million endowment gift from the Li Weibo Charitable Foundation in China and designed to build on the school’s already substantial accomplishments in the fields of gene therapy, RNA biology and RNAi technology in the development of novel therapeutics for a host of disorders. UMMS scientists are studying more than 20 rare diseases, including ALS, cystic fibrosis, Canavan disease, Rett syndrome, Huntington’s disease, fragile X syndrome, CDKL5 disorder and others.
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, said he and his colleagues work closely with patients and their families, a constant reminder of the urgency of their work in the lab.
“Canavan families are part of my family,” Gao said. “Every time I meet with patients and their family I am motivated to work harder and faster to get cures to them.”
Learn more in the broadcast and informational video, below:
Facebook Live video: https://www.facebook.com/UMassMed/videos/10156042510083361
A Rare Commitment to Rare Disease video: https://youtu.be/FNWuXG9vhsY