Recombinant AAV-directed gene therapy for type i glycogen storage diseases

Expert Opinion on Biological Therapy

Volumn 11, Issue 8, 2011, Pages 1011-1024

  Chou, Janice Y ^a   Mansfield, Brian C ^a,b  

^a EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^b Correlogic Systems Inc ^*  (United States)

Author keywords

AAV; endoplasmic reticulum; gene therapy; glucose homeostasis; glucose 6 phosphatase; glucose 6 phosphate transporter; glycogen storage disease; hepatic gene delivery

Indexed keywords

GLUCOSE 6 PHOSPHATASE; GLUCOSE 6 PHOSPHATASE ALPHA; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

DRUG EFFICACY; DRUG MECHANISM; GENE TARGETING; GENE THERAPY; GENETIC TRANSDUCTION; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 1A; GLYCOGEN STORAGE DISEASE TYPE 1B; HUMAN; IMMUNE RESPONSE; NONHUMAN; REVIEW; TREATMENT RESPONSE;

ANIMALS; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; GLYCOGEN STORAGE DISEASE TYPE I; HUMANS; RECOMBINATION, GENETIC;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 79959886898     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2011.578067     Document Type: Review

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