Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle-wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons. Here, we show that CjCas9 derived from Campylobacter jejuni can be used as a gene-editing tool to...

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Pubblicato in:Mol Ther
Autori principali: Koo, Taeyoung, Lu-Nguyen, Ngoc B., Malerba, Alberto, Kim, Eunji, Kim, Daesik, Cappellari, Ornella, Cho, Hee-Yeon, Dickson, George, Popplewell, Linda, Kim, Jin-Soo
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2018
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5986736/
https://ncbi.nlm.nih.gov/pubmed/29730196
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.03.018
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