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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)...
Autores principales: | Lee, Ciaran M., Flynn, Rowan, Hollywood, Jennifer A., Scallan, Martina F., Harrison, Patrick T. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc.
2012
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Materias: | |
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 |
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