Gene therapy for lysosomal storage disorders

Expert Opinion on Biological Therapy

Volumn 11, Issue 9, 2011, Pages 1153-1167

  Gritti, Angela ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

Author keywords

CNS; Gene delivery; Gene therapy; Lysosomal storage disorders; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; ALPHA LEVO IDURONIDASE; BETA N ACETYLHEXOSAMINIDASE; CEREBROSIDE SULFATASE; GENE PRODUCT; IDURONATE 2 SULFATASE; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; SOMATOMEDIN C; UNCLASSIFIED DRUG; VASCULOTROPIN 165; VIRUS VECTOR;

CELL TYPE; CENTRAL NERVOUS SYSTEM DISEASE; EX VIVO GENE TRANSFER; GANGLIOSIDOSIS; GENE EXPRESSION REGULATION; GENE THERAPY; GENOTOXICITY; GLYCOGEN STORAGE DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOGENICITY; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NEURAL STEM CELL TRANSPLANTATION; NIEMANN PICK DISEASE; NONHUMAN; PLURIPOTENT STEM CELL; REVIEW; RISK ASSESSMENT; SANDHOFF DISEASE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

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

EID: 80051510219     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.2011.582036     Document Type: Review

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