Article
Systemic in utero gene editing as a treatment for cystic fibrosis.
Proceedings of the National Academy of Sciences of the United States of America - 17 Jun 2025
Ricciardi Adele S, Barone Christina, Putman Rachael, Quijano Elias, Gupta Anisha, Nguyen Richard, Mandl Hanna, Piotrowski-Daspit Alexandra S, Lopez-Giraldez Francesc, Luks Valerie, Freedman-Weiss Mollie R, Farrelly James, Ahle Samantha, Lynn Anna Y, Glazer Peter M, Saltzman W Mark, Stitelman David H, Egan Marie E
Abstract excerpt
In utero gene editing has the potential to modify disease-causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a disease that arises from mutations in the CF transmembrane conductance regulator (CFTR) gene, there are signs of multiorgan disease affecting the function of the respiratory,...
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