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Newsweek: UMass Medical School develops gene therapy for rare neurodegenerative disease

Gao lab shows AAV gene therapy on path to clinical trial for Canavan disease

  In the Gao lab are, (from left,) Dominic Gessler, Rachel Epstein (seated), her sister, Jessica, and Guangping Gao, PhD.

In the Gao lab are, (from left,) Dominic Gessler, Rachel Epstein (seated), her sister, Jessica, and Guangping Gao, PhD.

Researchers in the lab of Guangping Gao, PhD, have developed a novel gene therapy that cures the rare, inherited neurodegenerative disorder Canavan disease in mice and is on track to be brought to clinical trial, according to an April 28 article in Newsweek.

“We have the therapy. We are ready,” said 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 at UMass Medical School. Gao, a global leader in the field of adeno-associated virus (AAV) gene therapy, told the magazine that his goal is to cure Canavan disease.

Dominic Gessler, a medical fellow leading the Canavan research in Gao’s lab, explained to Newsweek how the therapy works. It uses a virus, AAV, not known to cause disease, to deliver a healthy copy of a gene to the brain. Gessler told Newsweek he will never forget the first time he tested it a live mouse.

“He injected it into a young, sick mouse, hunched over with no control over its muscles,” wrote reporter Megan Scudellari, in the story. “When Gessler returned to assess the results, he couldn’t find the mouse. He worried he had mislabeled a cage. He hadn’t. The mouse was there, acting just as healthy as the control mice.”

Worcester’s Sherri Epstein, whose 17-year-old daughter Rachel has Canavan and who had previously took part in a gene therapy trial, has stayed abreast of  Gao’s research since he first opened his lab at UMMS in 2008. In the story, Epstein is described holding two of the mice from Gao’s lab, one of whom has been cured of Canavan, yet there is no discernable difference between the two.

Read the full Newsweek story here: Gene therapy might be the best, and perhaps only, chance at curing brain diseases.

Related links on UMassMedNow:
Horae Oriental endows Gene Therapy Center at UMass Medical School with $2M gift
Voyager Therapeutics targets novel gene therapies to combat diseases