Abstract:
:In retinitis pigmentosa, loss of cone photoreceptors leads to blindness, and preservation of cone function is a major therapeutic goal. However, cone loss is thought to occur as a secondary event resulting from degeneration of rod photoreceptors. Here we report a genome editing approach in which adeno-associated virus (AAV)-mediated CRISPR/Cas9 delivery to postmitotic photoreceptors is used to target the Nrl gene, encoding for Neural retina-specific leucine zipper protein, a rod fate determinant during photoreceptor development. Following Nrl disruption, rods gain partial features of cones and present with improved survival in the presence of mutations in rod-specific genes, consequently preventing secondary cone degeneration. In three different mouse models of retinal degeneration, the treatment substantially improves rod survival and preserves cone function. Our data suggest that CRISPR/Cas9-mediated NRL disruption in rods may be a promising treatment option for patients with retinitis pigmentosa.
journal_name
Nat Communjournal_title
Nature communicationsauthors
Yu W,Mookherjee S,Chaitankar V,Hiriyanna S,Kim JW,Brooks M,Ataeijannati Y,Sun X,Dong L,Li T,Swaroop A,Wu Zdoi
10.1038/ncomms14716subject
Has Abstractpub_date
2017-03-14 00:00:00pages
14716issn
2041-1723pii
ncomms14716journal_volume
8pub_type
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