Proof-of-Concept Trial Provides Hope for AAV Gene Therapies

Investigators at the University of Massachusetts Medical School and the University of Florida in Gainesville are reporting success in a Phase I proof-of-concept trial using gene therapy to treat a rare disease, with possible implications for treating a broad range of disorders. Investigators used a harmless adeno-associated virus to inject a “corrected” gene into nine patients to help their bodies manufacture a necessary protein, the medical school says in a statement. The patients have alpha-1 antitrypsin deficiency, a hereditary defect.
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