Gene therapy for the lysosomal storage disorders

Current Opinion in Molecular Therapeutics

Volumn 4, Issue 4, 2002, Pages 349-358

  Cabrera Salazar, Mario A ^a   Novelli, Enrico ^a   Barranger, John A ^a  

^a UNIVERSITY OF PITTSBURGH   (United States)

Author keywords

Animal models; BMT; Enzyme replacement; Gene therapy; Gene transfer; Lysosomal storage disorders

Indexed keywords

ALPHA GLUCOSIDASE; CD3 ANTIGEN; CEREBROSIDE SULFATASE; COMPLEMENTARY DNA; CYCLIN DEPENDENT KINASE; CYCLIN DEPENDENT KINASE INHIBITOR; CYCLINE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDE; GLYCOGEN; IMIGLUCERASE; LENTIVIRUS VECTOR; LYSOSOME ENZYME; RETROVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; BONE MARROW TRANSPLANTATION; CLINICAL FEATURE; CLINICAL TRIAL; DRUG EFFICACY; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LYSOSOME STORAGE DISEASE; MOUSE; NEUROPATHOLOGY; NONHUMAN; PATHOPHYSIOLOGY; RETROSPECTIVE STUDY; REVIEW; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BONE MARROW TRANSPLANTATION; DISEASE MODELS, ANIMAL; FABRY DISEASE; GAUCHER DISEASE; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; LYSOSOMAL STORAGE DISEASES; MICE; RABBITS; RETROVIRIDAE;

ADENO-ASSOCIATED VIRUS; ANIMALIA; LENTIVIRUS; UNIDENTIFIED RETROVIRUS;

EID: 0036705301     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

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