HGT: Scientists hope to use mRNA therapy or CRISPR gene editing techniques to treat cystic fibrosis.

11, 2020 /PRY/ -- In a recent study published in the international journal Human Gene Therapy entitled "Treating Cystic Fibrosis with mRNA and CRISPR", scientists from the Georgia Institute of Technology and others have revealed how mRNA therapy or CRISPR technology can be used to treat patients with cystic fibrosis.
Photo Source: University of Cambridge, researchers say the potential for using mRNA therapy or CRISPR gene editing techniques to treat cystic fibrosis (CF, Cystic Fibrosis) is not likely to be associated with pathogenic mutations in the patient's body, and that clinical trials related to cystic fibrosis currently show that agenotic gene therapy for cystic fibrosis appears to be feasible.
James Dahlman says that the treatment of cystic fibrosis by transporting mRNA to encode CFTR may work in any cystic fibrosis patient's body, which does not seem to depend on the mutations carried in the patient's body, while another potential treatment is to use mRNA to encode nucleases associated with gRNA, such as CRISPR-Cas9, and use it to edit DNA in targeted cells.
, of course, how to successfully use these methods remains a challenge for scientists, who first need to identify drug transport systems that can reach the skin cells of the lungs in low doses.
Finally, the researchers said that cystic fibrosis is the first disease target to study multiple vector platforms for human diseases, including rAAV and rAd, and the researchers are pleased that this new technology can be applied to the study of cystic fibrosis, especially in patients with cystic fibrosis who carry 5% of the mutations that are resistant to CFTR regulatory drugs;
() Original source: Alejandro Da Silva Sanchez et al. Treating Cystic Fibrosis with mRNA and CRISPR, Human Gene Therapy (2020). DOI: 10.1089/hum.2020.137.