Gene therapy for lysosomal storage disorders: a good start

Human Molecular Genetics

Volumn 25, Issue R1, 2016, Pages R65-R75

  Biffi, Alessandra ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

CHAPERONE; GLYCOPROTEIN; GLYCOSAMINOGLYCAN; LENTIVIRUS VECTOR;

BIOSYNTHESIS; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; COGNITION; ENDOCYTOSIS; ENZYME ACTIVITY; ENZYME REPLACEMENT; FIBROBLAST; GENE THERAPY; GENE TRANSFER; GLOBOID CELL LEUKODYSTROPHY; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HURLER SYNDROME; HYPERSENSITIVITY; IMMUNE DEFICIENCY; IMMUNOSUPPRESSIVE TREATMENT; LYSOSOME; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MORBIDITY; MORTALITY; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPATHOLOGY; OLIGODENDROGLIA; PHENOTYPE; REVIEW; SUBSTRATE REDUCTION THERAPY; LYSOSOMAL STORAGE DISEASES;

GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LYSOSOMAL STORAGE DISEASES;

EID: 85011779758     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv457     Document Type: Review

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