Gene transfer strategies for correction of lysosomal storage disorders

Acta Haematologica

Volumn 110, Issue 2-3, 2003, Pages 71-85

  D'Azzo, Alessandra ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Animal models; Gene therapy; Lysosomal enzymes; Lysosomal storage diseases; Viral vectors

Indexed keywords

ACID LIPASE; ACYLSPHINGOSINE DEACYLASE; ADENOVIRUS VECTOR; ALPHA LEVO FUCOSIDASE; ALPHA MANNOSIDASE; ALPHA N ACETYLGALACTOSAMINIDASE; ALPHA N ACETYLGLUCOSAMINIDASE; BETA GALACTOSIDASE; BETA GLUCOSIDASE; BETA GLUCURONIDASE; BETA MANNOSIDASE; BETA N ACETYLHEXOSAMINIDASE A; BETA N ACETYLHEXOSAMINIDASE B; CARBOXYPEPTIDASE C; CATHEPSIN K; CEREBROSIDE SULFATASE; CLN3 PROTEIN; GALACTOSYLCERAMIDASE; GLUCAN 1,4 ALPHA GLUCOSIDASE; HYALURONIDASE; HYDROLASE; IDURONATE 2 SULFATASE; LEVO IDURONIDASE; LYSOSOME ENZYME; MUCOLIPIDIN; N ACETYLGALACTOSAMINE 4 SULFATASE; N4 (BETA N ACETYLGLUCOSAMINYL)ASPARAGINASE; PALMITOYL PROTEIN THIOESTERASE; RECOMBINANT ENZYME; SPHINGOMYELIN PHOSPHODIESTERASE; UNCLASSIFIED DRUG; UNINDEXED DRUG;

BLINDNESS; CELL CULTURE; CELL FUNCTION; CELL TYPE; DISEASE SEVERITY; DNA MODIFICATION; ENZYME REPLACEMENT; ENZYME SUBSTRATE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GLYCOGEN STORAGE DISEASE; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LIPIDOSIS; LIPOFUSCINOSIS; LYSOSOME STORAGE DISEASE; MACROMOLECULE; MENTAL DEFICIENCY; METABOLITE; MORTALITY; MUCOLIPIDOSIS; MUCOPOLYSACCHARIDOSIS; NEUROLOGIC DISEASE; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL; REVIEW; STEM CELL TRANSPLANTATION; SYMPTOM;

ANIMALS; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LYSOSOMAL STORAGE DISEASES;

EID: 0242270777     PISSN: 00015792     EISSN: None     Source Type: Journal    
DOI: 10.1159/000072456     Document Type: Review

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