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. 2021 May;28(5):209-216.
doi: 10.1038/s41434-020-0168-2. Epub 2020 Jun 19.

Retinal gene therapy: an eye-opener of the 21st century

Affiliations

Retinal gene therapy: an eye-opener of the 21st century

Anne Louise Askou et al. Gene Ther. 2021 May.
No abstract available

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References

    1. High KA, Roncarolo MG. Gene therapy. N Engl J Med. 2019;381:455–64. - PubMed
    1. Trapani I, Auricchio A. Seeing the light after 25 years of retinal gene therapy. Trends Mol Med. 2018;24:669–81. - PubMed
    1. Garafalo AV, Cideciyan AV, Heon E, Sheplock R, Pearson A, WeiYang Yu C, et al. Progress in treating inherited retinal diseases: early subretinal gene therapy clinical trials and candidates for future initiatives. Prog Retin Eye Res. 2019:100827. [Online ahead of print].
    1. Trapani I, Auricchio A. Has retinal gene therapy come of age? From bench to bedside and back to bench. Hum Mol Genet. 2019;28:R108–18. - PubMed - PMC
    1. Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849–60. - PubMed - PMC

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