A single CRISPR-Cas9 deletion strategy that targets the majority of DMD patients restores dystrophin function in hiPSC-derived muscle cells

Mutations in DMD disrupt the reading frame, prevent dystrophin translation, and cause Duchenne muscular dystrophy (DMD). Here we describe a CRISPR/Cas9 platform applicable to 60% of DMD patient mutations. We applied the platform to DMD-derived hiPSCs where successful deletion and non-homologous end...

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Detalhes bibliográficos
Publicado no:Cell Stem Cell
Main Authors: Young, Courtney S., Hicks, Michael R., Ermolova, Natalia V., Nakano, Haruko, Jan, Majib, Younesi, Shahab, Karumbayaram, Saravanan, Kumagai-Cresse, Chino, Wang, Derek, Zack, Jerome A., Kohn, Donald B., Nakano, Atsushi, Nelson, Stanley F., Miceli, M. Carrie, Spencer, Melissa J., Pyle, April D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4826286/
https://ncbi.nlm.nih.gov/pubmed/26877224
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stem.2016.01.021
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