New gene-editing tool models and corrects cystic fibrosis mutations
Published in Diseases!.
Precision A3G editor reduces unintended nearby DNA changes in cells
Scientists have developed a more precise version of a gene-editing tool, showing that it can correct mutations that cause cystic fibrosis (CF) in cell models and also introduce CF-causing mutations into cells for research.
“We were able to introduce specific cystic-fibrosis mutations into human epithelial cells relevant to the disease, generating cell models that will improve our understanding,” Gang Bao, PhD, study co-senior author at Rice University, said in a university news story. “We were also able to reverse those mutations and show improved cellular functions using the same editor, demonstrating the level of pinpoint gene-editing control this technology now offers and the potential of base-pair editors to treat the disease.”