Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

Journal of Inherited Metabolic Disease

Volumn 35, Issue 5, 2012, Pages 847-857

  Roberts, Marie S ^a   Macauley, Shannon L ^a   Wong, Andrew M ^b   Yilmas, Denis ^b   Hohm, Sarah ^a   Cooper, Jonathan D ^b   Sands, Mark S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b KING'S COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLUCURONIDASE; CYSTAFOS; PALMITOYL PROTEIN THIOESTERASE; PALMITOYL PROTEIN THIOESTERASE 1; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUTOFLUORESCENCE; CONTROLLED STUDY; DRUG ACTIVITY; DRUG ADMINISTRATION ROUTE; DRUG BRAIN LEVEL; DRUG EFFICACY; ENZYME ASSAY; FEMALE; HISTOPATHOLOGY; IMMUNOHISTOCHEMISTRY; INTRACRANIAL DRUG ADMINISTRATION; LIFESPAN; MALE; MOTOR PERFORMANCE; MOUSE; NEURONAL CEROID LIPOFUSCINOSIS; NEURONAL CEROID LIPOFUSCINOSIS INFANTILE FINNISH TYPE; NONHUMAN; PRIMARY SOMATOSENSORY CORTEX; ROTAROD TEST; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BIOMIMETIC MATERIALS; BRAIN; CENTRAL NERVOUS SYSTEM; CYSTAPHOS; FEMALE; GENETIC THERAPY; MALE; MICE; MICE, INBRED C57BL; MOTOR ACTIVITY; NEURODEGENERATIVE DISEASES; NEURONAL CEROID-LIPOFUSCINOSES; THIOLESTER HYDROLASES;

EID: 84866731869     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-011-9446-x     Document Type: Article

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