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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...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5355895/ https://www.ncbi.nlm.nih.gov/pubmed/28291770 http://dx.doi.org/10.1038/ncomms14716 |