Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

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 ade...

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Dades bibliogràfiques
Publicat a:Nat Commun
Autors principals: Yu, Wenhan, Mookherjee, Suddhasil, Chaitankar, Vijender, Hiriyanna, Suja, Kim, Jung-Woong, Brooks, Matthew, Ataeijannati, Yasaman, Sun, Xun, Dong, Lijin, Li, Tiansen, Swaroop, Anand, Wu, Zhijian
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2017
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5355895/
https://ncbi.nlm.nih.gov/pubmed/28291770
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms14716
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