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Editorial
. 2024 Dec 2;14(12):a041728.
doi: 10.1101/cshperspect.a041728.

Retinal Disorders

José-Alain Sahel  1 , Eyal Banin  2 , Jean Bennett  3 , Jacque L Duncan  4 , Botond Roska  5
Affiliations
Editorial

Retinal Disorders

José-Alain Sahel et al. Cold Spring Harb Perspect Med. .

Abstract

Retinal disorders caused by genetic or environmental factors cause severe visual impairment and often result in blindness. The past ten years have seen rapid progress in our understanding of the biological basis of these conditions, as well as significant advances towards gene and cell-based therapies. Regulatory challenges remain, but there is reason to hope that creative approaches will lead to safe and effective breakthrough treatments for these conditions in the near future.

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References

    1. Audo I, Nassisi M, Zeitz C, Sahel JA. 2023. The extraordinary phenotypic and genetic variability of retinal and macular degenerations: The relevance to therapeutic developments. Cold Spring Harb Perspect Med a041652. 10.1101/cshperspect.a041652 - DOI - PMC - PubMed
    1. Awadh Hashem S, Georgiou M, Ali RR, Michaelides M. 2023. RPGR-Related retinopathy: Clinical features, molecular genetics, and gene replacement therapy. Cold Spring Harb Perspect Med 13: a041280. 10.1101/cshperspect.a041280 - DOI - PMC - PubMed
    1. Ayala A, Cheetham J, Durham T, Maguire M. 2023. The importance of natural history studies in inherited retinal diseases. Cold Spring Harb Perspect Med 13: a041297. 10.1101/cshperspect.a041297 - DOI - PMC - PubMed
    1. Bennett J. 2023. Overview of retinal gene therapy: Current status and future challenges. Cold Spring Harb Perspect Med 13: a041278. 10.1101/cshperspect.a041278 - DOI - PMC - PubMed
    1. Bennett J, Maguire AM. 2023. Lessons learned from the development of the first FDA-Approved gene therapy drug, voretigene neparvovec-rzyl. Cold Spring Harb Perspect Med 13: a041307. 10.1101/cshperspect.a041307 - DOI - PMC - PubMed

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