In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice

RATIONALE: Duchenne muscular dystrophy (DMD) is a severe inherited form of muscular dystrophy caused by mutations in the reading frame of the dystrophin gene disrupting its protein expression. Dystrophic cardiomyopathy is a leading cause of death in DMD patients and currently no effective treatment...

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Xehetasun bibliografikoak
Argitaratua izan da:Circ Res
Egile Nagusiak: Refaey, Mona El, Xu, Li, Gao, Yandi, Canan, Benjamin, Adesanya, T.M. Ayodele, Warner, Sarah C., Akagi, Keiko, Symer, David E., Mohler, Peter J., Ma, Jianjie, Janssen, Paul M.L., Han, Renzhi
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2017
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC5623072/
https://ncbi.nlm.nih.gov/pubmed/28790199
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1161/CIRCRESAHA.117.310996
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