Sneha Khedkar in The Scientist:
In 2019, the Food and Drug Administration approved Trikafta for patients with cystic fibrosis (CF) who have the most common disease-causing mutation. Trikafta targets the defective protein—a misfolded form of cystic fibrosis transmembrane conductance regulator (CFTR)—produced as a result of this mutation.1 However, people with other mutations in the CFTR gene, including those with variants that result in no proteins being made at all, cannot benefit from the drug. “Therefore, interest is being placed on genetic therapies,” said Daniel Siegwart, a chemist at the University of Texas Southwestern Medical Center.
Current gene therapy methods involve delivering mutation-correcting gene-editing tools directly to lung cells. However, such tools are often administered through inhalation and struggle to cross the thick, sticky mucus on their way to the cells.2 Now, in a paper published in Science, Siegwart and his team showed that lipid nanoparticles (LNPs) delivered intravenously successfully delivered gene-editing tools to all lung cell types, including stem cells.3 This approach enabled long-lasting gene correction in a mouse model of CF and in patient cells. The LNP-based system offers new therapeutic avenues for delivering gene-editing tools to specific tissues.
More here.
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