A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis (CF) — in stem cells from CF patients, a study shows.
The new approach has the ability to correct mutations without the need to excise the affected region, the researchers said.
The study, “CRISPR-Based Adenine Editors Correct Nonsense Mutations in a Cystic Fibrosis Organoid Biobank,” was published in the journal Cell Stem Cell.
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