Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

Eitemau Tebyg

• Hydrostatic Isolated Limb Perfusion with Adeno-associated Virus Vectors Enhances Correction of Skeletal Muscle in Pompe Disease
  gan: Sun, Baodong, et al.
  Cyhoeddwyd: (2010)
• Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease
  gan: Sun, Baodong, et al.
  Cyhoeddwyd: (2010)
• Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease
  gan: Zhang, Ping, et al.
  Cyhoeddwyd: (2012)
• Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice
  gan: Maga, John A., et al.
  Cyhoeddwyd: (2013)
• Enhanced Efficacy of an AAV Vector Encoding Chimeric, Highly-Secreted Acid α-glucosidase in Glycogen Storage Disease Type II
  gan: Sun, Baodong, et al.
  Cyhoeddwyd: (2006)