Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair

The use of zinc-finger nucleases (ZFNs) to permanently and precisely modify the human genome offers a potential alternative to cDNA-based gene therapy. The ΔF508 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene is observed in ∼70% of patients with cystic fibrosis (CF)...

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Detalles Bibliográficos
Autores principales: Lee, Ciaran M., Flynn, Rowan, Hollywood, Jennifer A., Scallan, Martina F., Harrison, Patrick T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2012
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3559198/
https://www.ncbi.nlm.nih.gov/pubmed/23514673
http://dx.doi.org/10.1089/biores.2012.0218

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3559198/
https://www.ncbi.nlm.nih.gov/pubmed/23514673
http://dx.doi.org/10.1089/biores.2012.0218

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