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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Main Authors: Svetlana A Smirnikhina, Ekaterina V Kondrateva, Elmira P Adilgereeva, Arina A Anuchina, Milyausha I Zaynitdinova, Yana S Slesarenko, Angelina S Ershova, Kirill D Ustinov, Matvei I Yasinovsky, Elena L Amelina, Ekaterina S Voronina, Valentina D Yakushina, Vyacheslav Yu Tabakov, Alexander V Lavrov
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2020-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0242094