Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations

Gene Therapy

Volumn 17, Issue 1, 2010, Pages 117-131

  Sun, X ^a   Pawlyk, B ^a   Xu, X ^a   Liu, X ^a   Bulgakov, O V ^a   Adamian, M ^a   Sandberg, M A ^a   Khani, S C ^b   Tan, M H ^c   Smith, A J ^c   Ali, R R ^c   Li, T ^a  

^a MASSACHUSETTS EYE AND EAR INFIRMARY   (United States)

^b SCHEPENS EYE RESEARCH INSTITUTE   (United States)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

AIPL1; Leber's congenital amaurosis; PDE6; Retinal degeneration

Indexed keywords

ARYL HYDROCARBON RECEPTOR INTERACTING PROTEIN LIKE 1; BINDING PROTEIN; PARVOVIRUS VECTOR; PHOSPHODIESTERASE; RHODOPSIN KINASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; ENZYME SYNTHESIS; GENE EXPRESSION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE TARGETING; GENETIC ASSOCIATION; MOUSE; MUTATIONAL ANALYSIS; NONHUMAN; PHENOTYPE; PHOTORECEPTOR; PRIORITY JOURNAL; PROMOTER REGION; RETINA CONE; RETINA DEGENERATION; RETINA ROD; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; CARRIER PROTEINS; G-PROTEIN-COUPLED RECEPTOR KINASE 1; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; MICE; OPTIC ATROPHY, HEREDITARY, LEBER; PHOSPHORIC DIESTER HYDROLASES; RETINAL CONE PHOTORECEPTOR CELLS; RETINAL DEGENERATION; RETINAL ROD PHOTORECEPTOR CELLS;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 74349104948     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.104     Document Type: Article

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