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GENE THERAPY

Gene therapy for retinal dystrophy

Nature Medicine volume 25, pages 198–199 (2019)Cite this article

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Counteracting splice defects in the CEP290 gene using RNA antisense oligonucleotides or Cas9-mediated gene editing is a therapeutic strategy for Leber congenital amaurosis type 10—a severe untreatable retinal dystrophy leading to childhood blindness.

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Fig. 1: Nucleic-acid-based therapies for inherited retinal degenerations.

References

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  2. Cideciyan, A. V. et al. Nat. Med. http://doi.org/10.1038/s41591-018-0295-0 (2019).

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Authors and Affiliations

  1. Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA

    José Alain Sahel

  2. Institut de la Vision, Sorbonne Université, INSERM, CNRS, Paris, France

    José Alain Sahel

  3. CHNO des Quinze-Vingts, DHU Sight Restore, INSERM-DGOS CIC 1423, Paris, France

    José Alain Sahel

  4. Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France

    Deniz Dalkara

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  1. José Alain Sahel
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  2. Deniz Dalkara
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Correspondence to José Alain Sahel.

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The authors declare no competing interests

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Sahel, J.A., Dalkara, D. Gene therapy for retinal dystrophy. Nat Med 25, 198–199 (2019). https://doi.org/10.1038/s41591-019-0346-1

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