CRISPR-based genome surgery for the treatment of autosomal dominant retinitis pigmentosa

OBJECTIVE: Develop a universal gene therapy to overcome the genetic heterogeneity in retinitis pigmentosa (RP) due to mutations in rhodopsin (RHO). DESIGN: Experimental study for a combination gene therapy that utilizes both gene ablation and gene replacement. PARTICIPANTS: This study includes two k...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Ophthalmology
Päätekijät: Tsai, Yi-Ting, Wu, Wen-Hsuan, Lee, Ting-Ting, Wu, Wei-Pu, Xu, Christine L., Park, Karen S., Cui, Xuan, Justus, Sally, Lin, Chyuan-Sheng, Jauregui, Ruben, Su, Pei-Yin, Tsang, Stephen H.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 2018
Aiheet:
Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC6109419/
https://ncbi.nlm.nih.gov/pubmed/29759820
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ophtha.2018.04.001
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