Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors

Pompe disease is a lysosomal storage disease caused by the absence of acid α-1,4 glucosidase (GAA). The pathophysiology of Pompe disease includes generalized myopathy of both cardiac and skeletal muscle. We sought to use recombinant adeno-associated virus (rAAV) vectors to deliver functional GAA gen...

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Veröffentlicht in:	Molecular therapy 2002-05, Vol.5 (5), p.571-578
Hauptverfasser:	Fraites, Thomas J., Schleissing, Mary R., Shanely, R.Andrew, Walter, Glenn A., Cloutier, Denise A., Zolotukhin, Irene, Pauly, Daniel F., Raben, Nina, Plotz, Paul H., Powers, Scott K., Kessler, Paul D., Byrne, Barry J.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adenoviruses alpha-Glucosidases - genetics alpha-Glucosidases - metabolism Animals Antibodies cardiovascular diseases Cardiovascular Diseases - metabolism Cardiovascular Diseases - therapy Dependovirus - genetics Disease Disease Models, Animal Enzymes Fibroblasts Fibroblasts - metabolism Gene Expression Regulation Gene therapy Genetic Therapy - methods Genetic Vectors Genomics Glycogen - metabolism glycogen storage disease type II Glycogen Storage Disease Type II - enzymology Glycogen Storage Disease Type II - genetics Glycogen Storage Disease Type II - therapy Homozygote Humans Immunoenzyme Techniques Infant lysosomal storage diseases Lysosomal Storage Diseases - metabolism Lysosomal Storage Diseases - therapy Mice Mice, Inbred BALB C Mice, Knockout Muscle, Skeletal - physiopathology musculoskeletal diseases Musculoskeletal system Myocardium - metabolism Proteins Transduction, Genetic Vectors (Biology) Viruses
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