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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Veröffentlicht in:	Nature communications Jg. 8; H. 1; S. 14716 - 15
Hauptverfasser:	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:	Journal Article
Sprache:	Englisch
Veröffentlicht:	London Nature Publishing Group UK 14.03.2017 Nature Publishing Group Nature Portfolio
Schlagworte:	38/1 38/70 42 42/109 45 45/23 45/70 59 59/5 631/208/4041/3196 692/699/3161/3175 Animals Basic-Leucine Zipper Transcription Factors - genetics Cell Survival - genetics CRISPR CRISPR-Cas Systems Dependovirus Disease Eye Proteins - genetics Gene Editing - methods Gene Knockdown Techniques Genes Genetic engineering Genomes Humanities and Social Sciences Kinases Mice multidisciplinary Mutation Neurosciences Photoreception Photoreceptors Retina Retinal Cone Photoreceptor Cells - cytology Retinal Cone Photoreceptor Cells - metabolism Retinal Degeneration - genetics Retinal Rod Photoreceptor Cells - cytology Retinal Rod Photoreceptor Cells - metabolism Retinitis Pigmentosa - genetics RNA polymerase Science Science (multidisciplinary) Visual acuity United States > US Maryland
ISSN:	2041-1723, 2041-1723
Online-Zugang:	Volltext
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