Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA

Cystic Fibrosis is an autosomal recessive disorder caused by mutations in the CFTR gene. CRISPR mediated, template-dependent homology-directed gene editing has been used to correct the most common mutation, c.1521_1523delCTT / p.Phe508del (F508del) which affects ~70% of individuals, but the efficien...

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Detalles Bibliográficos
Autores principales: Sanz, David J., Hollywood, Jennifer A., Scallan, Martina F., Harrison, Patrick T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5581164/
https://www.ncbi.nlm.nih.gov/pubmed/28863137
http://dx.doi.org/10.1371/journal.pone.0184009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5581164/
https://www.ncbi.nlm.nih.gov/pubmed/28863137
http://dx.doi.org/10.1371/journal.pone.0184009

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