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P.F508del editing in cells from cystic fibrosis patients

Development of genome editing methods created new opportunities for the development of etiology-based therapies of hereditary diseases. Here, we demonstrate that CRISPR/Cas9 can correct p.F508del mutation in the CFTR gene in the CFTE29o- cells and induced pluripotent stem cells (iPSCs) derived from...

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Bibliografski detalji
Izdano u:PLoS One
Glavni autori: Smirnikhina, Svetlana A., Kondrateva, Ekaterina V., Adilgereeva, Elmira P., Anuchina, Arina A., Zaynitdinova, Milyausha I., Slesarenko, Yana S., Ershova, Angelina S., Ustinov, Kirill D., Yasinovsky, Matvei I., Amelina, Elena L., Voronina, Ekaterina S., Yakushina, Valentina D., Tabakov, Vyacheslav Yu., Lavrov, Alexander V.
Format: Artigo
Jezik:Inglês
Izdano: Public Library of Science 2020
Teme:
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7657551/
https://ncbi.nlm.nih.gov/pubmed/33175893
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0242094
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