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CRISPR corrects CF genetic mutations in patient-derived organoids

Two mutations in the CFTR gene linked to cystic fibrosis were corrected in patient-derived cells using CRISPR/Cas9 gene editing, researchers reported in Nature Communications. The technology restored CFTR function in cells carrying either the wild-type or mutated versions of CFTR, and in lung cells and mini-intestines grown from CF patients' intestinal epithelial cells, while leaving non-mutated DNA unaffected.

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