Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism

Volumn 107, Issue 1-2, 2012, Pages 213-221

  Hu, Jie ^b   Lu, Jui Yun ^a,b   Wong, Andrew M S ^c   Hynan, Linda S ^b   Birnbaum, Shari G ^b   Yilmaz, Denis S ^c   Streit, Barbara M ^c   Lenartowicz, Ewelina M ^c   Thompson, Thomas C M ^c   Cooper, Jonathan D ^c   Hofmann, Sandra L ^a,b  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^b UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^c KING'S COLLEGE LONDON   (United Kingdom)

Author keywords

Batten disease; Enzyme replacement therapy; Infantile neuronal ceroid lipofuscinosis; Lysosomal storage disorder

Indexed keywords

CD68 ANTIGEN; ENZYME ANTIBODY; GLIAL FIBRILLARY ACIDIC PROTEIN; PALMITOYL PROTEIN THIOESTERASE; RECOMBINANT PALMITOYL PROTEIN THIOESTERASE; UNCLASSIFIED DRUG;

ACINAR CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; AUTOFLUORESCENCE; BRAIN; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DEATH; DRUG MEGADOSE; ENZYME LINKED IMMUNOSORBENT ASSAY; ENZYME REPLACEMENT; FEMALE; KIDNEY TUBULE CELL; KNOCKOUT MOUSE; LIVER; LUNG; LYSOSOME STORAGE DISEASE; MACROPHAGE; MALE; MOTOR DYSFUNCTION; MOTOR PERFORMANCE; MOUSE; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPATHOLOGY; NONHUMAN; OUTCOME ASSESSMENT; PANCREAS; PRIORITY JOURNAL; SMALL INTESTINE; SPLEEN; SURVIVAL; THALAMUS; TREATMENT DURATION;

ANIMALS; BRAIN; DISEASE MODELS, ANIMAL; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; LYSOSOMES; MALE; MICE; MICE, KNOCKOUT; MOTOR ACTIVITY; NEURONAL CEROID-LIPOFUSCINOSES; RECOMBINANT PROTEINS; ROTAROD PERFORMANCE TEST; THIOLESTER HYDROLASES; VISCERA;

ANIMALIA; MUS;

EID: 84866171062     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2012.05.009     Document Type: Article

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