Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa

Human Molecular Genetics

Volumn 23, Issue 2, 2014, Pages 514-523

  Wert, Katherine J ^a,b,c,d   Sancho Pelluz, Javier ^a,b,d,e   Tsang, Stephen H ^a,b,d  

^a Och Spine at New York Presbyterian Hospitals   (United States)

^b NewYork Presbyterian Hospital   (United States)

^c Institute of Human Nutrition College of Physicians and Surgeons Columbia University ^*  (United States)

^d New York Presbyterian Hospital   (United States)

^e UNIVERSIDAD CATÓLICA DE VALENCIA SAN VICENTE MÁRTIR   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

AAV SEROTYPE 4; AAV SEROTYPE 8; BETA ACTIN; PHOSPHODIESTERASE VI; PHOSPHODIESTERASE VI ALPHA; RED FLUORESCENT PROTEIN; RHODOPSIN; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY PRODUCTION; ARTICLE; CELL SURVIVAL; CONTROLLED STUDY; DRUG EFFICACY; EARLY INTERVENTION; ELECTRORETINOGRAM; EXTRACELLULAR MATRIX; GENE MUTATION; HUMAN; MOUSE; NONHUMAN; PHOTORECEPTOR CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; RETINA DEGENERATION; RETINA ROD; RETINITIS PIGMENTOSA; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VISION; VISUAL FIELD;

ANIMALS; CYCLIC NUCLEOTIDE PHOSPHODIESTERASES, TYPE 6; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; EYE PROTEINS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED C57BL; RETINAL PIGMENT EPITHELIUM; RETINAL ROD PHOTORECEPTOR CELLS; RETINITIS PIGMENTOSA;

EID: 84898826569     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddt452     Document Type: Article

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