Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model

Science Translational Medicine

Volumn 8, Issue 326, 2016, Pages

  Alam, Md Suhail ^a   Getz, Michelle ^a   Haldar, Kasturi ^a  

^a UNIVERSITY OF NOTRE DAME   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HISTONE DEACETYLASE INHIBITOR; MACROGOL; VORINOSTAT; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; BETA CYCLODEXTRIN DERIVATIVE; HYDROXAMIC ACID; MACROGOL DERIVATIVE; MESSENGER RNA;

ACETYLATION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD BRAIN BARRIER; CELL CULTURE; CEREBELLUM DISEASE; CONTROLLED STUDY; DRUG DOSE REDUCTION; DRUG EFFICACY; FEMALE; LIFESPAN; MALE; MOUSE; NERVE DEGENERATION; NERVOUS SYSTEM INFLAMMATION; NEUROLOGIC DISEASE; NIEMANN PICK DISEASE; NONHUMAN; PRIORITY JOURNAL; PURKINJE CELL; SYMPTOMATOLOGY; SYSTEMIC DISEASE; ANIMAL; BLOOD; CHEMISTRY; COMPLICATION; DISEASE COURSE; DISEASE MODEL; DRUG EFFECTS; FIBROBLAST; GENETICS; INFLAMMATION; LIVER; METABOLISM; MUTATION; NIEMANN-PICK DISEASE, TYPE C; PATHOLOGY; SPLEEN; SURVIVAL ANALYSIS;

ANIMALS; BETA-CYCLODEXTRINS; BLOOD-BRAIN BARRIER; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; FIBROBLASTS; HISTONE DEACETYLASE INHIBITORS; HYDROXAMIC ACIDS; INFLAMMATION; LIVER; MICE; MUTATION; NERVE DEGENERATION; NIEMANN-PICK DISEASE, TYPE C; POLYETHYLENE GLYCOLS; PURKINJE CELLS; RNA, MESSENGER; SPLEEN; SURVIVAL ANALYSIS;

EID: 84975748727     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.aad9407     Document Type: Article

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