AAVbased

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Treatment of glutaric aciduria type I (GA-I) by intracerebroventricular administration of GCDH. Credit: Institute of Zoology, Chinese Academy of Sciences, China Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation in the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated at 1/100,000, with approximately 75,000 cases worldwide. Patients …

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria Read More »

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria

Treatment of glutaric aciduria type I (GA-I) by intracerebroventricular administration of GCDH. Credit: Institute of Zoology, Chinese Academy of Sciences, China Glutaric aciduria type I (GA-I) is an inherited disease caused by a mutation in the glutaryl-CoA dehydrogenase (GCDH) gene. The global incidence of GA-I is estimated at 1/100,000, with approximately 75,000 cases worldwide. Patients …

Promising study suggests AAV-based gene therapy can treat type I glutaric aciduria Read More »