Treating Cystic Fibrosis with mRNA and CRISPR.

ABSTRACT: Less than 20% of the protein coding genome is thought to be targetable using small molecules. mRNA therapies are not limited in the same way since in theory, they can silence or edit any gene by encoding CRISPR nucleases, or alternatively, produce any missing protein. Yet not all mRNA therapies are equally likely to succeed. Over the past several years, an increasing number of clinical trials with siRNA- and antisense oligonucleotide-based drugs have revealed three key concepts that will likely extend to mRNA therapies delivered by nonviral systems. First, scientists have come to understand that some genes make better targets for RNA therapies than others. Second, scientists have learned that the type and position of chemical modifications made to an RNA drug can alter its therapeutic wind

SUBMITTER: Da Silva Sanchez A 

PROVIDER: S-EPMC7495921 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature