Cystic fibrosis affects the lungs, but delivering gene therapies to the faulty cells is challenging SCIENCE PHOTO LIBRARY/Getty Images 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 at the University of Texas Southwestern Medical Center. Read more Advertisement “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 .
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. Get the most essential health and fitness news in your inbox every Saturday. A possible solution is to deliver the molecular tools for gene editing through the blood, packaged in lipid nanoparticles that slip past immune barriers.
Similar nanoparticles have already been used in more than a billion , and other teams have in this way. However, lipid nanoparti.
