CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by edi...

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Bibliografski detalji
Izdano u:Sci Adv
Glavni autori: Min, Yi-Li, Li, Hui, Rodriguez-Caycedo, Cristina, Mireault, Alex A., Huang, Jian, Shelton, John M., McAnally, John R., Amoasii, Leonela, Mammen, Pradeep P. A., Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Jezik:Inglês
Izdano: American Association for the Advancement of Science 2019
Teme:
Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6402849/
https://ncbi.nlm.nih.gov/pubmed/30854433
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aav4324
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