Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction

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 usin...

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書誌詳細
出版年:Mol Ther
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2020
主題:
Original Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7335734/
https://ncbi.nlm.nih.gov/pubmed/32402246
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.04.021
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