Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Human Gene Therapy

Volumn 29, Issue 1, 2018, Pages 42-50

  Zhang, Wei ^a   Li, Linjing ^a   Su, Qin ^a   Gao, Guangping ^a   Khanna, Hemant ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

cilia; ciliopathies; gene therapy; LCA; photoreceptor; retina

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; COMPLEMENTARY DNA; MINICEP290 PROTEIN; PROTEIN; UNCLASSIFIED DRUG; CEP290 PROTEIN, HUMAN; TUMOR ANTIGEN; TUMOR PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL FUNCTION; CELL STRUCTURE; CELL SURVIVAL; CILIUM; CONTROLLED STUDY; EMBRYO; EMBRYO CELL; FIBROBLAST; IN VITRO STUDY; LEBER CONGENITAL AMAUROSIS; MOUSE; NONHUMAN; PHOTORECEPTOR; RETINA DEGENERATION; SUBRETINAL DRUG ADMINISTRATION; TREATMENT OUTCOME; VIRAL GENE THERAPY; ADMINISTRATION AND DOSAGE; ANIMAL; CELL CULTURE; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN;

ANIMALS; ANTIGENS, NEOPLASM; CELLS, CULTURED; CILIA; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LEBER CONGENITAL AMAUROSIS; MICE; NEOPLASM PROTEINS;

EID: 85040600794     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2017.049     Document Type: Article

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