Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease

Evaluation of Hydrodynamic Limb Vein Injections in Nonhuman Primates

(2010) Julia O. Hegge et al.   HUMAN GENE THERAPY

Improved Induction of Immune Tolerance to Factor IX by Hepatic AAV-8 Gene Transfer

(2009) Mario Cooper et al.   HUMAN GENE THERAPY

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Evaluation of Vascular Delivery Methodologies to Enhance rAAV6-mediated Gene Transfer to Canine Striated Musculature

(2009) Paul Gregorevic et al.   MOLECULAR THERAPY

Ability of Adeno-Associated Virus Serotype 8-Mediated Hepatic Expression of Acid α-Glucosidase to Correct the Biochemical and Motor Function Deficits of Presymptomatic and Symptomatic Pompe Mice

(2008) Robin J. Ziegler et al.   HUMAN GENE THERAPY

Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

(2008) Baodong Sun et al.   MOLECULAR THERAPY