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...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Mol Ther
Hauptverfasser: Koo, Taeyoung, Lu-Nguyen, Ngoc B., Malerba, Alberto, Kim, Eunji, Kim, Daesik, Cappellari, Ornella, Cho, Hee-Yeon, Dickson, George, Popplewell, Linda, Kim, Jin-Soo
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
Schlagworte:
Original Article
Online Zugang: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
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!

Ähnliche Einträge