Emerging therapies for neurodegenerative lysosomal storage disorders - From concept to reality

Journal of Inherited Metabolic Disease

Volumn 34, Issue 5, 2011, Pages 1003-1012

  Hemsley, Kim M ^a   Hopwood, John J ^a  

^a WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLUCURONIDASE; CHAPERONE; GENISTEIN; IBUPROFEN; IDURONATE 2 SULFATASE; INDOMETACIN; LENTIVIRUS VECTOR; LEVO IDURONIDASE; LYSOSOME ENZYME; MIGLUSTAT; PARVOVIRUS VECTOR; PHYTOESTROGEN; PYRIMETHAMINE; RETROVIRUS VECTOR; SOMATOMEDIN B RECEPTOR;

ASPARTYLGLYCOSAMINURIA; BIOACCUMULATION; BLOOD BRAIN BARRIER; CALORIC RESTRICTION; CELL BASED GENE THERAPY; DEGENERATIVE DISEASE; DOSE RESPONSE; DRUG HALF LIFE; ENZYME ACTIVITY; ENZYME INHIBITION; ENZYME REPLACEMENT; FUCOSIDOSIS; GAUCHER DISEASE; GENE MUTATION; GENETIC CODE; GLOBOID CELL LEUKODYSTROPHY; GM1 GANGLIOSIDOSIS; GM2 GANGLIOSIDOSIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LATE INFANTILE NEURONAL CEROID LIPOFUSCINOSIS; LOW DRUG DOSE; LYSOSOME STORAGE DISEASE; MANNOSIDOSIS; METACHROMATIC LEUKODYSTROPHY; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 7; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPATHOLOGY; NIEMANN PICK DISEASE; NONHUMAN; REVIEW; SANDHOFF DISEASE; SANFILIPPO SYNDROME TYPE A; SANFILIPPO SYNDROME TYPE B; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; CELL TRANSPLANTATION; CONCEPT FORMATION; ENZYME REPLACEMENT THERAPY; EVIDENCE-BASED PRACTICE; GENE THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES, NERVOUS SYSTEM; MODELS, BIOLOGICAL; NEURODEGENERATIVE DISEASES; THERAPIES, INVESTIGATIONAL;

EID: 82955233827     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-011-9341-5     Document Type: Review

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