Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis

Biochimica et Biophysica Acta - Molecular Basis of Disease

Volumn 1832, Issue 10, 2013, Pages 1784-1792

  Bonten, Erik J ^a   Yogalingam, Gouri ^a,b   Hu, Huimin ^a   Gomero, Elida ^a   Van de Vlekkert, Diantha ^a   D'Azzo, Alessandra ^a  

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

^b BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

AAV; Chaperone mediated therapy; Gene therapy; Lysosomal storage disease; NEU1; Sialidosis

Indexed keywords

RECOMBINANT ADENOVIRUS PROTEIN CATHEPSIN A; SIALIDASE; UNCLASSIFIED DRUG; VIRUS VACCINE;

AMINO ACID SUBSTITUTION; ARTICLE; CELL TYPE; CHERRY RED SPOT; EMBRYO; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; HUMAN CELL; KIDNEY; MUCOLIPIDOSIS TYPE 1; MUTANT; MUTATION; MYOCLONUS; PRIORITY JOURNAL;

AAV; BMEF; BONE MARROW EXTRACELLULAR FLUID; CHAPERONE-MEDIATED THERAPY; ENZYME REPLACEMENT THERAPY; ERT; FACE; FLUOROPHORE-ASSISTED CARBOHYDRATE ELECTROPHORESIS; GALACTOSIALIDOSIS; GENE THERAPY; GS; IHC; IMMUNOHISTOCHEMISTRY; KNOCKOUT; KNOCKOUT-TRANSGENIC; KO; KO/TG; LSD; LYSOSOMAL STORAGE DISEASE; NEU1; PCT; PHARMACOLOGIC-CHAPERONE THERAPY; PPCA; PROTECTIVE PROTEIN/CATHEPSIN A; SCAAV; SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS; SIALIDOSIS; WILD-TYPE; WT;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; MICE; MICE, KNOCKOUT; MOLECULAR CHAPERONES; MUCOLIPIDOSES; RECOMBINATION, GENETIC;

EID: 84880581088     PISSN: 09254439     EISSN: 1879260X     Source Type: Journal    
DOI: 10.1016/j.bbadis.2013.06.002     Document Type: Article

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