In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter...

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Detalles Bibliográficos
Autores principales: Bakondi, Benjamin, Lv, Wenjian, Lu, Bin, Jones, Melissa K, Tsai, Yuchun, Kim, Kevin J, Levy, Rachelle, Akhtar, Aslam Abbasi, Breunig, Joshua J, Svendsen, Clive N, Wang, Shaomei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786918/
https://www.ncbi.nlm.nih.gov/pubmed/26666451
http://dx.doi.org/10.1038/mt.2015.220

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786918/
https://www.ncbi.nlm.nih.gov/pubmed/26666451
http://dx.doi.org/10.1038/mt.2015.220

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