Engineers sharpen gene-editing tools to target cystic fibrosis
Medical Xpress
February 23, 2026
AI-Generated Deep Dive Summary
Engineers at the University of Pennsylvania and Rice University have made significant strides in enhancing gene-editing technology, achieving unprecedented precision by targeting individual genetic "base pairs." This breakthrough could revolutionize treatments for genetic diseases, particularly cystic fibrosis (CF). Current therapies often address symptoms rather than root causes, but this new method offers a more targeted approach. Researchers believe it could lead to therapies with better outcomes than existing options.
Cystic fibrosis is a genetic disorder caused by mutations in the CFTR gene, affecting lung function and leading to severe respiratory issues. While treatments like mucus-thinning drugs and airway clearance techniques help manage symptoms, they do not address the underlying genetic defect. This new gene-editing tool could potentially correct specific mutations in the CFTR gene, offering a more direct solution.
The technology builds upon CRISPR systems, known for their precision in editing DNA. The researchers have refined the system to target single base pairs with higher accuracy, reducing off-target effects—a common concern with gene-editing tools. This improvement not only enhances safety but also makes it more reliable for treating genetic diseases like CF. Unlike previous methods that may cause unintended edits
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Originally published on Medical Xpress on 2/23/2026