In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

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• 作者：Mert Yanik^a ; ¹ ; Brigitte Mü ; ller^a ; ¹ ; Fei Song^a ; ¹ ; Jacqueline Gall^a ; ¹ ; Franziska Wagner^a ; ¹ ; Wolfgang Wende^b ; ¹ ; Birgit Lorenz^a ; ¹ ; Knut Stieger^a ; ¹ ; ^{knut.stieger@uniklinikum-giessen.de}
• 关键词：Genome editing ; In vivo ; Retina ; CRISPR-Cas ; TALEN ; AAV ; Endonucleases ; NHEJ ; HDR ; MMEJ
• 刊名：Progress in Retinal and Eye Research
• 出版年：2017
• 出版时间：January 2017
• 年：2017
• 卷：56
• 期：Complete
• 页码：1-18
• 全文大小：3637 K
• 卷排序：56

文摘

In vivo genome editing in the retina has to challenge efficient gene transfer, DNA repair and absence of toxicity. CRISPR-Cas and TALEN represent highly specific tools for the induction of double strand breaks. Use of viral vectors, nanoparticals and direct protein transfer are potential ways to introduce items into photoreceptors. In depth knowledge about the DNA repair pathways in the retina is key to successful in vivo genome editing applications. Initial studies in mouse photoreceptors indicate that the DNA repair machinery is active albeit at comparably low levels.