Biologists in The Netherlands have shown for the first time that a new type of CRISPR gene editing can safely cure cystic fibrosis in stem cells derived from patients.
Called base editing, the technique involves altering the Cas-part so that it does not create a cut, like the older CRISPR/Cas 9 technique, but still detects the mutation and enables the mutation to be repaired on site.
Maarten Geurts, of the Hubrecht Institute, and Eyleen de Poel, from UMC Utrecht, say the new version is a more effective genome editing tool that can be safely and effectively applied in human stem cells.
The results of their study, which used "mini-guts" from a biobank of intestinal organoids, created using the stem cells of cystic fibrosis (CF) patients, are published in Cell Stem Cell.
Guerts said: "CF is caused by a mistake, a mutation, in the CFTR-gene leading to malfunctioning of the gene. As a consequence, the mucus in many organs, including the lungs, is less hydrated, resulting in mucus build-up and organ failure. With the new base-editing technique the mutation in the CFTR-gene can be detected and repaired without creating further damage in the genome."
De Poel said their research represents a significant step towards genetic repair of diseases in patients.
“However, a big question that remains is how to deliver the CRISPR-enzyme to the appropriate organs in the patient,” she said. “Cystic fibrosis might also not be the most suitable disease to treat with CRISPR, as many organs are affected by the disease.”
The team says further research is needed before the base-editor can be used for clinical application.
Guerts MH, de Poel E, Amatngalim GD et al. CRISPR-based adenine editors correct nonsense mutations in a cystic fibrosis organoid biobank. Cell Stem Cell 20 February 2020
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