Promising study suggests that AAV-based gene therapy can treat glutaric aciduria type I
Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation of the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated to be 1/100,000, with around 75,000 cases worldwide.
Nov 29, 2022
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