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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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| 出版年: | Mol Ther |
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| 主要な著者: | , , , , , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
American Society of Gene & Cell Therapy
2020
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| 主題: | |
| 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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