Recent advances in gene therapy for lysosomal storage disorders

Application of Clinical Genetics

Volumn 8, Issue , 2015, Pages 157-169

  Rastall, David P W ^a   Amalfitano, Andrea ^a,b  

^a Michigan State University ^*  (United States)

^b MICHIGAN STATE UNIVERSITY   (United States)

Author keywords

Adeno associated virus; Adenovirus; Blood brain barrier; Clinical trials; Gene therapy; Human trials; Lentivirus; Lysosomal storage disease

Indexed keywords

ADENOVIRUS VECTOR; CRYOPYRIN; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; TOLL LIKE RECEPTOR 2; TOLL LIKE RECEPTOR 9;

ADAPTIVE IMMUNITY; ARTICLE; BLOOD BRAIN BARRIER; CELLULAR IMMUNITY; DISEASE MODEL; ENZYME ACTIVITY; ENZYME REPLACEMENT; ENZYME SYNTHESIS; EX VIVO GENE TRANSFER; GAA GENE; GENE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE VECTOR; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUMORAL IMMUNITY; HURLER SYNDROME; IMMUNOMODULATION; IMMUNOSUPPRESSIVE TREATMENT; INNATE IMMUNITY; INTRACRANIAL DRUG ADMINISTRATION; INTRANASAL GENE DELIVERY; LIVER DIRECTED GENE THERAPY; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; NEUROPATHIC LYSOSOME STORAGE DISEASE; NONHUMAN; PPT1 GENE; PROMOTER REGION; PROTEIN EXPRESSION; SEROTYPE; SGSH GENE; STORAGE DISEASE; TRANSIENT IMMUNOSUPPRESSION; TREATMENT INDICATION;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; LENTIVIRUS;

EID: 84936933033     PISSN: None     EISSN: 1178704X     Source Type: Journal    
DOI: 10.2147/TACG.S57682     Document Type: Article

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