Neonatal Aav Gene Therapy In A Viable Mouse Model Of Combined Methylmalonic Acidemia And Homocystinuria, Cblc Type, Is Comparable To Chronic Hydroxocobalamin Administration

Stefanos A. Koutsoukos,Jennifer L. Sloan, Kelsey Murphy,Madeline Arnold,Nathan Achilly, Gene Elliot,Patricia Zerfas, Victoria Hoffmann,Charles P. Venditti

MOLECULAR THERAPY(2021)

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