UConn Health and Connecticut Children’s have made history by dosing the first patient in a clinical trial using gene editing to treat glycogen storage disease type 1a (GSD1a).

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This groundbreaking trial marks the third “first-in-human” trial for GSD1a, a rare inherited metabolic disorder, conducted by the research team.

The therapy involves permanent gene editing to correct a genetic error in liver cells, offering hope for patients with GSD and other rare diseases.

“This therapy marks a groundbreaking achievement for the treatment of GSD and similar rare diseases,” said Juan C. Salazar, MD, Chair of the Department of Pediatrics at the UConn School of Medicine and Physician-in-Chief at Connecticut Children’s.

“This milestone represents a beacon of hope for patients and their families worldwide, showcasing out commitment to advancing healthcare and saving lives.”

Led by a dedicated team of researchers, this trial represents a significant advancement in gene-based therapies and rare disease research, with the potential to revolutionize treatment for genetic conditions in the future.

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