Gene therapies for cystic fibrosis have previously struggled to reach the faulty lung cells, but a new approach has succeeded in achieving long-lasting modifications in mice
By Christa Lesté-Lasserre
13 June 2024
Cystic fibrosis affects the lungs, but delivering gene therapies to the faulty cells is challenging
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A CRISPR gene-editing therapy has the potential to offer an effective, long-lasting treatment for cystic fibrosis after overcoming a major challenge that held back previous genetic therapies.
The approach has succeeded in editing DNA in hard-to-reach lung stem cells in mice, with modifications that endured for at least 22 months – essentially the animals’ entire lives, says Daniel Siegwart at the University of Texas Southwestern Medical Center.
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“I was falling out of my chair with amazement after seeing a few months of persistence,” he says. “This is exciting news for potential treatment of a variety of lung ailments.”
Cystic fibrosis is caused by genetic mutations that lead to sticky mucus building up in the lungs and digestive system.
Scientists have previously developed gene-editing technologies to modify the DNA in the faulty lung cells. But getting the therapeutic agents into those cells is a challenge due to the mucus and other defences that have evolved to keep pathogens out of the lungs, says Siegwart.