Replacement gene therapy with a Human RPGRIP1 sequence slows photoreceptor degeneration in a murine model of leber congenital amaurosis

Human Gene Therapy

Volumn 21, Issue 8, 2010, Pages 993-1004

  Pawlyk, Basil S ^a   Bulgakov, Oleg V ^a,c   Liu, Xiaoqing ^a   Xu, Xiaoyun ^a   Adamian, Michael ^a   Sun, Xun ^a,c   Khani, Shahrokh C ^b   Berson, Eliot L ^a   Sandberg, Michael A ^a   Li, Tiansen ^a,c  

^a MASSACHUSETTS EYE AND EAR INFIRMARY   (United States)

^b SCHEPENS EYE RESEARCH INSTITUTE   (United States)

^c NATIONAL EYE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; RHODOPSIN KINASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; ELECTRORETINOGRAM; GENE; GENE EXPRESSION; GENE REPLACEMENT THERAPY; GENE SEQUENCE; GENE TARGETING; LEBER CONGENITAL AMAUROSIS; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; ORGAN PRESERVATION; PHOTORECEPTOR; PROMOTER REGION; PROTEIN DEGRADATION; RETINA DEGENERATION; RPGRIP1 GENE; SEROTYPE; TRANSGENE;

ANIMALS; DISEASE MODELS, ANIMAL; ELECTRORETINOGRAPHY; G-PROTEIN-COUPLED RECEPTOR KINASE 1; GENE THERAPY; GENETIC VECTORS; HUMANS; LEBER CONGENITAL AMAUROSIS; MICE; MICE, INBRED C57BL; PHOTORECEPTOR CONNECTING CILIUM; PROMOTER REGIONS, GENETIC; PROTEINS; RETINAL DEGENERATION;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

EID: 77955332214     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2009.218     Document Type: Article

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