Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism

Volumn 114, Issue 2, 2015, Pages 281-293

  Vuillemenot, Brian R ^a   Kennedy, Derek ^a   Cooper, Jonathan D ^b   Wong, Andrew M S ^b   Sri, Sarmi ^b   Doeleman, Thom ^b   Katz, Martin L ^c   Coates, Joan R ^c   Johnson, Gayle C ^c   Reed, Randall P ^d   Adams, Eric L ^d   Butt, Mark T ^e   Musson, Donald G ^a   Henshaw, Joshua ^a   Keve, Steve ^a   Cahayag, Rhea ^a   Tsuruda, Laurie S ^a   O'Neill, Charles A ^a  

^a BIOMARIN PHARMACEUTICAL INC   (United States)

^b KING'S COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY OF MISSOURI   (United States)

^d Northern Biomedical Research Inc   (United States)

^e Tox Path Specialists LLC   (United States)

Author keywords

Batten disease; CLN2; Intracerebroventricular; Intrathecal; Neuronal ceroid lipofuscinosis; Tripeptidyl peptidase 1

Indexed keywords

ENZYME ANTIBODY; RECOMBINANT ENZYME; RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE I; TRIPEPTIDYL PEPTIDASE I; UNCLASSIFIED DRUG; AMINOPEPTIDASE; ANTIBODY; DIPEPTIDYL PEPTIDASE; RECOMBINANT PROTEIN; SERINE PROTEINASE; TRIPEPTIDYL-PEPTIDASE 1;

ANIMAL EXPERIMENT; ANIMAL MODEL; AREA UNDER THE CURVE; ARTICLE; CENTRAL NERVOUS SYSTEM; CEREBROSPINAL FLUID; CHRONIC DRUG ADMINISTRATION; CONTROLLED STUDY; DACHSHUND (DOG); DISEASE COURSE; DRUG BLOOD LEVEL; DRUG DISTRIBUTION; DRUG HALF LIFE; DRUG SAFETY; EDEMA; ENCEPHALITIS; ENZYME REPLACEMENT; GENOTYPE; HISTOPATHOLOGY; HYPERESTHESIA; IMMUNOGENICITY; LIPOFUSCINOSIS; LYSOSOME STORAGE DISEASE; MAXIMUM PLASMA CONCENTRATION; MORPHOLOGY; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPATHOLOGY; NONHUMAN; PLASMA CONCENTRATION-TIME CURVE; PLEOCYTOSIS; PRESERVATION; PRIORITY JOURNAL; SPINE DISEASE; TIME TO MAXIMUM PLASMA CONCENTRATION; WILD TYPE; ANIMAL; BLOOD; BRAIN; DOG; DRUG SCREENING; IMMUNOLOGY; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; NEURONAL CEROID-LIPOFUSCINOSES; PATHOLOGY; ULTRASTRUCTURE;

ANIMALIA;

AMINOPEPTIDASES; ANIMALS; ANTIBODIES; BRAIN; DIPEPTIDYL-PEPTIDASES AND TRIPEPTIDYL-PEPTIDASES; DISEASE PROGRESSION; DOGS; DRUG EVALUATION, PRECLINICAL; ENZYME REPLACEMENT THERAPY; GENOTYPE; INFUSIONS, INTRAVENTRICULAR; NEURONAL CEROID-LIPOFUSCINOSES; RECOMBINANT PROTEINS; SERINE PROTEASES;

EID: 84921689811     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2014.09.004     Document Type: Article

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