Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy

Ophthalmology

Volumn 123, Issue 7, 2016, Pages 1606-1620

  Weleber, Richard G ^a   Pennesi, Mark E ^a   Wilson, David J ^a   Kaushal, Shalesh ^b   Erker, Laura R ^a   Jensen, Lauren ^a   McBride, Maureen T ^a   Flotte, Terence R ^b   Humphries, Margaret ^b   Calcedo, Roberto ^d   Hauswirth, William W ^c   Chulay, Jeffrey D ^e   Stout, J Timothy ^a  

^a OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^b UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^c UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e APPLIED GENETIC TECHNOLOGIES CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DORZOLAMIDE; HYDROLASE; ISOMEROHYDROLASE RPE65; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; TIMOLOL; UNCLASSIFIED DRUG; CIS TRANS ISOMERASE; RETINOID ISOMEROHYDROLASE;

ABNORMAL SENSATION; ADULT; ANTIBODY TITER; ARTICLE; BACKACHE; CHILD; CHILDHOOD DISEASE; CLINICAL ARTICLE; CONJUNCTIVAL HEMORRHAGE; CONTROLLED STUDY; DARK ADAPTATION; DRUG EFFICACY; DRUG SAFETY; ELECTRORETINOGRAPHY; EYE DISEASE; EYE IRRITATION; EYE PAIN; FEMALE; FOLLOW UP; HEADACHE; HUMAN; HYPEREMIA; HYPERTENSION; INTRAOCULAR PRESSURE; INTRAOCULAR PRESSURE ABNORMALITY; LEBER CONGENITAL AMAUROSIS; MALE; MULTICENTER STUDY; NONCARDIAC CHEST PAIN; OPTICAL COHERENCE TOMOGRAPHY; OUTCOME ASSESSMENT; PERIMETRY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PHOTOPIC VISION; PHOTOPSIA; PRESCHOOL CHILD; PRIORITY JOURNAL; QUALITY OF LIFE; QUALITY OF LIFE ASSESSMENT; RETINA DYSTROPHY; RETINOTOMY; SCHOOL CHILD; SEVERE EARLY CHILDHOOD ONSET RETINAL DYSTROPHY; SUBRETINAL DRUG ADMINISTRATION; VIRAL GENE THERAPY; VISION; VISUAL ACUITY; VISUAL FIELD; VITRECTOMY; CLINICAL TRIAL; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; INTRAOCULAR DRUG ADMINISTRATION; PATHOPHYSIOLOGY; PHYSIOLOGY; PROCEDURES; RETINAL DEGENERATION; YOUNG ADULT;

ADULT; CHILD; CIS-TRANS-ISOMERASES; DEPENDOVIRUS; ELECTRORETINOGRAPHY; FEMALE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INJECTIONS, INTRAOCULAR; LEBER CONGENITAL AMAUROSIS; MALE; QUALITY OF LIFE; RETINAL DEGENERATION; VISUAL ACUITY; VISUAL FIELDS; YOUNG ADULT;

EID: 84963812408     PISSN: 01616420     EISSN: 15494713     Source Type: Journal    
DOI: 10.1016/j.ophtha.2016.03.003     Document Type: Article

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