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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7657551/
https://www.ncbi.nlm.nih.gov/pubmed/33175893
http://dx.doi.org/10.1371/journal.pone.0242094

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