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    Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

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    Authors
    Pennesi, Mark E.
    Weleber, Richard G.
    Yang, Paul
    Whitebirch, Chris
    Thean, Beverly
    Flotte, Terence R.
    Humphries, Margaret
    Chegarnov, Elvira
    Beasley, Kathleen N.
    Stout, J. Timothy
    Chulay, Jeffrey D.
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    UMass Chan Affiliations
    Gene Therapy Center
    Department of Pediatrics
    Document Type
    Journal Article
    Publication Date
    2018-07-24
    Keywords
    AAV
    Leber congenital amaurosis
    RPE65
    gene therapy
    retinal degeneration
    Enzymes and Coenzymes
    Eye Diseases
    Genetic Phenomena
    Genetics and Genomics
    Pediatrics
    Therapeutics
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    Link to Full Text
    https://doi.org/10.1089/hum.2018.014
    Abstract
    Previously, results at 2 years after subretinal injection of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in eight adults and four children with retinal degeneration caused by RPE65 mutations were reported. Now, results at 5 years after treatment in 11 of these subjects are reported. Subjects received a subretinal injection of rAAV2-CB-hRPE65 in the poorer-seeing eye, at either of two dose levels, and were followed for 5 years after treatment. The primary safety outcomes were ocular and non-ocular adverse events. Efficacy outcomes included changes in best corrected visual acuity, static perimetry hill of vision measurements for the central 30 degrees (V30), and total (VTOT) visual field and kinetic perimetry visual field area. The only adverse events reported during years 3, 4, and 5 were minor intercurrent illnesses. Pediatric subjects had improvement in visual acuity and static perimetry in the treated eye, sometimes with a smaller improvement in the untreated eye, during the first 2 years of the study that persisted during years 3-5, with no consistent changes in kinetic perimetry during the study. Most adult subjects had no consistent changes in visual acuity or static perimetry during the study. Three adult subjects with markedly abnormal baseline kinetic visual field area had improvement in the treated eye during the first 1-2 years after treatment, but the absolute magnitude of the improvement was small and was not sustained at subsequent visits. There were no clinically significant adverse events. Visual acuity and static perimetry testing results suggest that treating patients at a younger age is associated with better visual function outcomes during 5 years after treatment.
    Source

    Hum Gene Ther. 2018 Jul 24. doi: 10.1089/hum.2018.014. [Epub ahead of print] Link to article on publisher's site

    DOI
    10.1089/hum.2018.014
    Permanent Link to this Item
    http://hdl.handle.net/20.500.14038/43652
    PubMed ID
    29869534
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    Link to Article in PubMed

    ae974a485f413a2113503eed53cd6c53
    10.1089/hum.2018.014
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