CRISPR/Cas9 variant technology corrects cystic fibrosis of cultured human stem cells

Cystic fibrosis (CF) is one of the most common genetic diseases in the world, with serious consequences for patients

Correct CF mutation

The researchers successfully corrected the mutations that cause CF in human intestinal organoids

Safer than CRISPR/Cas9

Prime editing is an updated version of the more widely known gene editing technology CRISPR/Cas9

swelling

The mutation that causes CF is located in the CFTR channel, which is present in cells of various organs including the lung

Treatment of genetic diseases

Now, researchers have shown that the mutations that cause CF can be safely corrected, and the clinical application has taken another step forward

Hans Clevers is the team leader of the Hubrecht Institute for Developmental Biology and Stem Cells and the Princess Máxima Children’s Oncology Center

Maarten H Geurts, Eyleen de Poel, Cayetano Pleguezuelos-Manzano, Rurika Oka, Léo Carrillo, Amanda Andersson-Rolf, Matteo Boretto, Jesse E Brunsveld, Ruben van Boxtel, Jeffrey M Beekman, Hans Clevers.