AAV9-Mediated Gene Therapy for Infantile-Onset Pompe’s Disease

Abstract excerpt

Summary Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid α-glucosidase (GAA) (dose, 1.2×10 14 vector genomes per kilogram of body weight). One patient was withdrawn from the study and subsequently died. The other patients had improvement in cardiac outcomes and...

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