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Von Gierke Disease (GSD1)

Research & Development

The world’s first gene therapy trial in Glycogen Storage Disease.

First ever gene therapy for GSD

On 24 July 2018 a patient received the first ever gene therapy for glycogen storage disease (GSD). It was developed by the team led by Dr. David Weinstein, director of the Glycogen Storage Disease Program at Connecticut Children’s Medical Center and UConn Health. 

Jerrod has lived with the consequences of his rare and potentially deadly genetic liver disorder, GSD1a, since birth and his diagnosis at 9 months old.

As part of a new clinical trial Jerrod received the novel gene therapy via an intravenous infusion. It is hoped that it will significantly improve his disease. The research team will test the therapy’s safety and effectiveness six weeks into treatment, when Jerrod will try to stop taking cornstarch and they will see how his body responds.

Video by University of Connecticut.