Pioneering Therapies Reshaping Healthcare

Gene editing, particularly using the CRISPR technology, has recently gained significant attention in the field of medicine. The US has approved the world’s first gene-editing therapy, which has the potential to transform the medical industry by correcting mutations that cause various diseases. The approved treatment, known as “exa-cell,” targets sickle cell disease, and there are approximately 280 gene-editing therapies in development, with a focus on hereditary diseases like cystic fibrosis and certain cancers. <h2>Gene Editing for Cystic Fibrosis</h2> Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene editing, specifically CRISPR genome editing, holds promise as a potential therapy for CF. The CRISPR gene editing tools, including a “guide” that locates the mutated sequence in the CFTR gene, have shown potential in correcting the mutations that cause CF. <a href="https://medium.com/science-and-medicine-in-everyday-language/pioneering-therapies-reshaping-healthcare-1ba57097a3e1">Website</a>