Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

Biochemical Society Transactions

Volumn 38, Issue 6, 2010, Pages 1484-1488

  Wong, Andrew M S ^a   Rahim, Ahad A ^b   Waddington, Simon N ^b   Cooper, Jonathan D ^a  

^a KING'S COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Batten disease; Chaperone therapy; Enzyme replacement therapy; Gene therapy; Neuronal ceroid lipofuscinosis; Stem cell therapy

Indexed keywords

ADENOVIRUS VECTOR; LYSOSOME ENZYME; MEMBRANE PROTEIN; RECOMBINANT ENZYME; RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE 1; RECOMBINANT PALMITOYL PROTEIN THIOESTERASE 1; RECOMBINANT PROTEIN CLN2; SOMATOMEDIN B RECEPTOR; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ADENOVIRUS 2; CLINICAL TRIAL; CONFERENCE PAPER; DRUG EFFICACY; DRUG FATALITY; ENDOCYTOSIS; ENZYME REPLACEMENT; EPILEPTIC STATE; FABRY DISEASE; GAUCHER DISEASE; GENE THERAPY; GM1 GANGLIOSIDOSIS; HUMAN; HYPOTHALAMUS VENTROMEDIAL NUCLEUS; INFANTILE NEURONAL CEROID LIPOFUSCINOSIS; MISSENSE MUTATION; MUCOPOLYSACCHARIDOSIS; NEURONAL CEROID LIPOFUSCINOSIS; NEURONAL CEROID LIPOFUSCINOSIS LATE INFANTILE TYPE; NONHUMAN; PRIORITY JOURNAL; PROTEIN TARGETING; SCID MOUSE; SPINAL MUSCULAR ATROPHY; STEM CELL TRANSPLANTATION; THERAPY EFFECT; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; CHILD; ENDOCYTOSIS; ENZYME REPLACEMENT THERAPY; GENE THERAPY; HUMANS; LYSOSOMES; MEMBRANE PROTEINS; MICE; MOLECULAR CHAPERONES; MUTATION; NEURONAL CEROID-LIPOFUSCINOSES; RECEPTOR, IGF TYPE 2; STEM CELL TRANSPLANTATION;

EID: 78649738728     PISSN: 03005127     EISSN: 14708752     Source Type: Journal    
DOI: 10.1042/BST0381484     Document Type: Conference Paper

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