Molecular pathologies of and enzyme replacement therapies for lysosomal diseases

CNS and Neurological Disorders - Drug Targets

Volumn 5, Issue 4, 2006, Pages 401-413

  Sakuraba, Hitoshi ^a,b   Sawada, Makoto ^a,c,d   Matsuzawa, Fumiko ^a,e   Aikawa, Sei Ichi ^a,e   Chiba, Yasunori ^a,f   Jigami, Yoshifumi ^a,f   Itoh, Kohji ^a,g  

^a JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

^b TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE   (Japan)

^c NAGOYA UNIVERSITY   (Japan)

^d Tissue Targeting Japan Inc   (Japan)

^e Celestar Lexico Sciences Inc   (Japan)

^f NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGY AIST   (Japan)

^g UNIVERSITY OF TOKUSHIMA   (Japan)

Author keywords

Drug delivery; Enzyme replacement therapy; Fabry disease; Lysosomal disease; Lysosomal enzyme; Protein structure; Sandhoff disease; Tay Sachs disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALGUCOSIDASE ALPHA; ALPHA GALACTOSIDASE; ALPHA GLUCOSIDASE; BETA GALACTOSIDASE; BETA N ACETYLHEXOSAMINIDASE A; BETA N ACETYLHEXOSAMINIDASE B; ENZYME INHIBITOR; GALSULFASE; GANGLIOSIDE GM2; GLUCOSYLCERAMIDASE; HYGROMYCIN; IDURONATE 2 SULFATASE; IDUROSULFASE; IMIGLUCERASE; LARONIDASE; LEVO IDURONIDASE; LYSOSOME ENZYME; N ACETYLGALACTOSAMINE 4 SULFATASE; N ACETYLGLUCOSAMINE; NEOMYCIN; OLIGOSACCHARIDE; RECOMBINANT ENZYME; RETROVIRUS VECTOR; SPHINGOMYELIN PHOSPHODIESTERASE; UNCLASSIFIED DRUG;

CLINICAL TRIAL; DISEASE ASSOCIATION; DRUG BINDING SITE; DRUG DESIGN; ENZYME REPLACEMENT; ENZYME SYNTHESIS; FABRY DISEASE; GAUCHER DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; MAROTEAUX LAMY SYNDROME; MICROGLIA; MOLECULAR BIOLOGY; NEUROLOGIC DISEASE; NIEMANN PICK DISEASE; NONHUMAN; PATHOPHYSIOLOGY; PROTEIN STRUCTURE; PROTEIN SYNTHESIS INHIBITION; REVIEW; SANDHOFF DISEASE; TAY SACHS DISEASE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BETA-N-ACETYLHEXOSAMINIDASE; CULTURE MEDIA, CONDITIONED; ENZYMES; G(M2) GANGLIOSIDE; HUMANS; LYSOSOMAL STORAGE DISEASES, NERVOUS SYSTEM; LYSOSOMES; RECOMBINANT FUSION PROTEINS; SANDHOFF DISEASE; TAY-SACHS DISEASE;

EID: 33746996417     PISSN: 18715273     EISSN: None     Source Type: Journal    
DOI: 10.2174/187152706777950738     Document Type: Review

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