CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype

Gene Therapy

Volumn 21, Issue 7, 2014, Pages 662-672

  Burnight, E R ^a   Wiley, L A ^a   Drack, A V ^a   Braun, T A ^a,b   Anfinson, K R ^a   Kaalberg, E E ^a   Halder, J A ^a   Affatigato, L M ^a   Mullins, R F ^a   Stone, E M ^a,c   Tucker, B A ^a  

^a UNIVERSITY OF IOWA   (United States)

^b IA United States   (United States)

^c UNIVERSITY OF IOWA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CEP290 PROTEIN; COMPLEMENTARY DNA; LENTIVIRUS VECTOR; MICROTUBULE PROTEIN; UNCLASSIFIED DRUG; CEP290 PROTEIN, HUMAN; TUMOR ANTIGEN; TUMOR PROTEIN;

ANIMAL CELL; ARTICLE; CELL DIFFERENTIATION; CELL FUNCTION; CILIOGENESIS; CONTROLLED STUDY; EUKARYOTIC FLAGELLUM; FIBROBLAST CULTURE; GENE CONSTRUCT; GENE EXPRESSION; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO GENE TRANSFER; LEBER CONGENITAL AMAUROSIS; MOUSE; NONHUMAN; PHENOTYPE; PHOTORECEPTOR; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; RETINA; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SKIN FIBROBLAST; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WESTERN BLOTTING; WILD TYPE; ANIMAL; CELL CULTURE; DISEASE MODEL; FIBROBLAST; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; INDUCED PLURIPOTENT STEM CELL; LENTIVIRINAE; METABOLISM; PATHOLOGY; PHARMACOLOGY; PHOTORECEPTOR CELL; TRANSPLANTATION;

ANIMALS; ANTIGENS, NEOPLASM; CELLS, CULTURED; CILIA; DISEASE MODELS, ANIMAL; FIBROBLASTS; GENETIC VECTORS; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LEBER CONGENITAL AMAUROSIS; LENTIVIRUS; MICE; NEOPLASM PROTEINS; PHOTORECEPTOR CELLS; RETINA; TRANSDUCTION, GENETIC;

EID: 84903885678     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2014.39     Document Type: Article

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