Scientists hope to use CRISPR-Cas technology to cull disease-causing gene mutations

The results are expected to open up a new perspective for the development of new treatments for cystic fibrosis, which is caused by a special gene mutation that produces cystic fibrosis transmembrane conduction regulators (Cftrs) that have abnormal functions that affect the function of multiple organs, especially the lungs, in which researchers used the CRISPR-Cas gene editing system to permanently edit at least two types of mutations that trigger cystic fibrosisGiulia Maule, a doctoral student at the, said: 'We have developed a gene editing strategy based on CRISPR-Cas that permanently removes two mutations that induce disease, and CRISPR-Cas works like a surgical knife, which can cut off the mutated gene element with great accuracy, and that can be used to repair the patient's body-derived organ with high precision, allowing only the sequence of mutations to be targeted and retain unaffectedthe study, researchers did not use animal models, and they used organs that develop from patients' body cells to conduct the study, which may help them effectively verify the effectiveness and accuracy of this new strategyCystic fibrosis is an invisible disease because it does not have an external indicator, but it can cause great damage to the patient's life, mainly causing problems in the lungs and digestive system, which is inherited from parents; in Italy, one in 25 people is carrier of cystic fibrosis, which means that parents have a one in a quarter chance of inheriting the disease to future generations, about 6,000 patients in Italy, and 200 new cases a yearoriginal origins: Giulia Maule, Antonio Casini, Claudia Montagna, et alAllele Prex of Splicing mutations in cystic fibrosis through CasCas12a genome, Nature Communications (2019)DOI: 10.1038/s41467-019-11454-9original title: Nat Commun: New Hope for Cystic Fibrosis Treatment Scientists Hope to Use CRISPR-Cas Technology to reject pathogenic gene mutations