Article
Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction.
Molecular therapy : the journal of the American Society of Gene Therapy - 8 Jul 2020
Suzuki Shingo, Crane Ana M, Anirudhan Varada, Barillà Cristina, Matthias Nadine, Randell Scott H, Rab Andras, Sorscher Eric J, Kerschner Jenny L, Yin Shiyi, Harris Ann, Mendel Matthew, Kim Kenneth, Zhang Lei, Conway Anthony, Davis Brian R
Abstract excerpt
There is a strong rationale to consider future cell therapeutic approaches for cystic fibrosis (CF) in which autologous proximal airway basal stem cells, corrected for CFTR mutations, are transplanted into the patient's lungs. We assessed the possibility of editing the CFTR locus in these cells using zinc-finger nucleases and have pursued two approaches. The first, mutation-specific correction, is a...
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