Sustained expression and safety of human GNE in normal mice after gene transfer based on AAV8 systemic delivery

Neuromuscular Disorders

Volumn 22, Issue 11, 2012, Pages 1015-1024

  Mitrani Rosenbaum, Stella ^a   Yakovlev, Lena ^a   Becker Cohen, Michal ^a   Telem, Michal ^a   Elbaz, Moran ^a   Yanay, Nurit ^a   Yotvat, Hagit ^a   Ben Shlomo, Uri ^a   Harazi, Avi ^a   Fellig, Yakov ^a   Argov, Zohar ^a   Sela, Ilan ^a  

^a HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

Author keywords

AAV8; Gene therapy; GNE myopathy; Systemic injection

Indexed keywords

COMPLEMENTARY DNA; GNE PROTEIN; MANNOSAMINE; MESSENGER RNA; PROTEIN; SIALIC ACID; UNCLASSIFIED DRUG; URIDINE DIPHOSPHATE N ACETYLGLUCOSAMINE; URIDINE DIPHOSPHATE N ACETYLGLUCOSAMINE 2 EPIMERASE; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; GENE; GENE EXPRESSION; GENE FUNCTION; GENE MUTATION; GENE OVEREXPRESSION; GENE THERAPY; GENE TRANSFER; GNE GENE; MOUSE; MUSCLE CELL; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SKELETAL MUSCLE; VIRUS PARTICLE; WILD TYPE;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED C57BL; MULTIENZYME COMPLEXES; MUSCLE, SKELETAL; MUTATION; MYOSITIS, INCLUSION BODY; SAFETY; TRANSFER (PSYCHOLOGY);

EID: 84868131658     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2012.03.013     Document Type: Article

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