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CRISPR restores expression in models of vision loss

  1. Megan Cully
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Inherited retinal dystrophies can present as early as infancy and lead to severe or complete vision loss. They are also often monogenic and therefore amenable to gene therapy. Editas Medicine has now developed a CRISPR–Cas9-based approach that can restore gene expression in mouse and non-human primate (NHP) models of the retinal dystrophy Leber congenital amaurosis type 10 (LCA10), thereby potentially moving gene editing with CRISPR platforms one step closer to the clinic.

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Nature Reviews Drug Discovery 18, 174 (2019)

doi: https://doi.org/10.1038/d41573-019-00016-9

References

  1. Maeder, M. L. et al. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10. Nat. Med. https://doi.org/10.1038/s41591-018-0327-9 (2019)

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