Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Myotonic dystrophy type 1 (DM1) is caused by a CTG nucleotide repeat expansion within the 3′ UTR of the Dystrophia Myotonica protein kinase gene. In this study, we explored therapeutic genome editing using CRISPR/Cas9 via targeted deletion of expanded CTG repeats and targeted insertion of polyadenyl...

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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Wang, Yanlin, Hao, Lei, Wang, Hongcai, Santostefano, Katherine, Thapa, Arjun, Cleary, John, Li, Hui, Guo, Xiuming, Terada, Naohiro, Ashizawa, Tetsuo, Xia, Guangbin
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society of Gene & Cell Therapy 2018
Konular:
Original Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6225032/
https://ncbi.nlm.nih.gov/pubmed/30274788
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.09.003
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