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Researchers at Hans Clevers (Hubrecht Institute) corrected the mutation that caused cystic fibrosis in 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
Start-up 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
DOI
https://doi.
Article title
Evaluating CRISPR-based Prime Editing for cancer modeling and CFTR repair in organoids