Cell and gene-based therapies for the lysosomal storage diseases

Current Gene Therapy

Volumn 6, Issue 2, 2006, Pages 227-241

  Hodges, Bradley L ^a   Cheng, Seng H ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

Central nervous system; Gene transfer; Immune tolerance; Liver; Lysosomal enzymes; Non viral vectors; Skeletal muscle; Stem cells; Viral

Indexed keywords

ADENOVIRUS VECTOR; ALPHA N ACETYLGLUCOSAMINIDASE; ARYLSULFATASE; BETA GLUCURONIDASE; CYCLOSPORIN; GALACTOSYLCERAMIDASE; GLUCAN 1,4 ALPHA GLUCOSIDASE; GLYCOSAMINOGLYCAN; IMMUNOSUPPRESSIVE AGENT; LEVO IDURONIDASE; LYSOSOME ENZYME; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PARVOVIRUS VECTOR; PLASMID DNA; RAPAMYCIN; RETROVIRUS VECTOR; RNA; SPHINGOMYELIN PHOSPHODIESTERASE; TRANSACTIVATOR PROTEIN;

ADOPTIVE IMMUNOTHERAPY; BONE MARROW TRANSPLANTATION; BRAIN; CELL FUNCTION; CELL ORGANELLE; DNA MODIFICATION; ENZYME ACTIVITY; ENZYME DEGRADATION; ENZYME REPLACEMENT; ENZYME SUBSTRATE; ENZYME SYNTHESIS; FABRY DISEASE; GAUCHER DISEASE; GENE CASSETTE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; GENETIC TRANSCRIPTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; IMMUNITY; IMMUNOLOGICAL TOLERANCE; INCIDENCE; LIVER; LYSOSOME; LYSOSOME STORAGE DISEASE; MESENCHYME CELL; MUSCLE TISSUE; NEURAL STEM CELL; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PALLIATIVE THERAPY; PREVALENCE; REVIEW; RNA INTERFERENCE; SKELETAL MUSCLE; TARGET ORGAN; UMBILICAL CORD; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; CELL TRANSPLANTATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; IMMUNE TOLERANCE; LYSOSOMAL STORAGE DISEASES; ORGAN SPECIFICITY; TRANSCRIPTION, GENETIC;

ADENOVIRIDAE; PARVOVIRUS;

EID: 33646195657     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/156652306776359522     Document Type: Review

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