Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases

Acta Paediatrica, International Journal of Paediatrics

Volumn 101, Issue 7, 2012, Pages 692-701

  Tomanin, Rosella ^a   Zanetti, Alessandra ^a   Zaccariotto, Eva ^a   D'Avanzo, Francesca ^a   Bellettato, Cinzia M ^a   Scarpa, Maurizio ^a  

^a UNIVERSITY OF PADOVA   (Italy)

Author keywords

Gene therapy; Lysosomal storage disorders; Mendelian diseases

Indexed keywords

ADENOVIRUS VECTOR; HERPESVIRUS VECTOR; IDURONATE 2 SULFATASE; LENTIVIRUS VECTOR; LIPID; NAKED DNA; PARVOVIRUS VECTOR; POLYMER; VIRUS VECTOR;

BRAIN BLOOD FLOW; CLINICAL TRIAL (TOPIC); ELECTRO GENE TRANSFER; ENZYME REPLACEMENT; FABRY DISEASE; GALACTOSIALIDOSIS; GAUCHER DISEASE; GENE GUN; GENE REPLACEMENT THERAPY; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; GLYCOGEN STORAGE DISEASE TYPE 2; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HYDRODYNAMICS; IMMUNOGENICITY; LYSOSOME STORAGE DISEASE; MEDICAL ETHICS; MENDELIAN DISEASE; METACHROMATIC LEUKODYSTROPHY; MITOSIS; MOLONEY LEUKEMIA ONCOVIRUS; MUCOPOLYSACCHARIDOSIS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; REVIEW; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM;

GENE THERAPY; GENETIC VECTORS; HUMANS; LYSOSOMAL STORAGE DISEASES;

EID: 84861798890     PISSN: 08035253     EISSN: 16512227     Source Type: Journal    
DOI: 10.1111/j.1651-2227.2012.02674.x     Document Type: Review

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