Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

Science Translational Medicine

Volumn 7, Issue 276, 2015, Pages

  Vite, Charles H ^a   Bagel, Jessica H ^a   Swain, Gary P ^a   Prociuk, Maria ^a   Sikora, Tracey U ^a   Stein, Veronika M ^a,g   O'Donnell, Patricia ^a   Ruane, Therese ^a   Ward, Sarah ^a   Crooks, Alexandra ^a   Li, Su ^a,h   Mauldin, Elizabeth ^a   Stellar, Susan ^b   De Meulder, Marc ^c   Kao, Mark L ^b   Ory, Daniel S ^d   Davidson, Cristin ^e   Vanier, Marie T ^f   Walkley, Steven U ^e  

^a UNIVERSITY OF PENNSYLVANIA SCHOOL OF VETERINARY MEDICINE   (United States)

^b JANSSEN RESEARCH AND DEVELOPMENT   (United States)

^c JANSSEN RESEARCH AND DEVELOPMENT   (Belgium)

^d WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

^f UNIVERSITÉ LYON 1   (France)

^g UNIVERSITY OF VETERINARY MEDICINE HANNOVER   (Germany)

^h KANSAS STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; CHOLESTEROL; GANGLIOSIDE; SPHINGOLIPID; 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN; ALANINE AMINOTRANSFERASE; BETA CYCLODEXTRIN DERIVATIVE; CALBINDIN; GANGLIOSIDE GM2;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOACCUMULATION; CELL LOSS; CEREBELLUM DISEASE; COHORT ANALYSIS; CONTROLLED STUDY; DISEASE COURSE; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG SAFETY; FEMALE; LUNG TOXICITY; MALE; NIEMANN PICK DISEASE; NONHUMAN; PRIORITY JOURNAL; PROSPECTIVE STUDY; PURKINJE CELL; AGING; ANIMAL; ATAXIA; AUDITORY THRESHOLD; BLOOD; CAT; CELL DEATH; CISTERNA MAGNA; COMPLICATION; FLUORESCENT ANTIBODY TECHNIQUE; INFLAMMATION; LIVER; LIVER DISEASE; LUNG; METABOLISM; NIEMANN-PICK DISEASE, TYPE C; PATHOLOGY; PATHOPHYSIOLOGY; STAINING; SUBCUTANEOUS DRUG ADMINISTRATION; SURVIVAL ANALYSIS;

AGING; ALANINE TRANSAMINASE; ANIMALS; ATAXIA; AUDITORY THRESHOLD; BETA-CYCLODEXTRINS; CALBINDINS; CATS; CELL DEATH; CISTERNA MAGNA; FLUORESCENT ANTIBODY TECHNIQUE; G(M2) GANGLIOSIDE; INFLAMMATION; INJECTIONS, SUBCUTANEOUS; LIVER; LIVER DISEASES; LUNG; NIEMANN-PICK DISEASE, TYPE C; PURKINJE CELLS; STAINING AND LABELING; SURVIVAL ANALYSIS;

EID: 84923923825     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3010101     Document Type: Article

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