囊性纤维化,mRNA疗法,CRISPR基因编辑技术 z-bo 2020-10-16 335

　　In the article, researchers use mRNA therapy or CRISPR gene editing technology to treat cystic fibrosis (CF) that does not seem to be related to pathogenic mutations in patients. He said it could be treated. At present, the results of clinical trials related to cystic fibrosis indicate that genotype-agnostic gene therapy for cystic fibrosis seems feasible. James Dahlman said that treatment of cystic fibrosis by transporting mRNA encoding CFTR may play a role in patients with cystic fibrosis. One possible treatment method is to use mRNA to encode gRNA-related nucleases (such as CRISPR-Cas9) and use it to edit the DNA of target cells.

　　Of course, how to use these methods well is still a challenge for scientists. First, researchers need to identify a drug delivery system that can reach lung epithelial cells in low doses. Finally, the researchers say that cystic fibrosis is the first disease target of multiple carrier platforms for studying human diseases. These vector platforms include rAAV and rAd. Researchers are very happy that this new technology can be used for cystic fibrosis, especially the study of cystic fibrosis. 5% of cystic fibrosis patients have mutations that are resistant to CFTR modulators. Later researchers will continue to conduct detailed studies to discover various new therapies that can effectively treat cystic fibrosis.