Improving transgene expression and CRISPR-Cas9 efficiency with molecular engineering-based molecules.

ABSTRACT: As a novel and robust gene-editing tool, the Clustered Regularly Interspaced Short Palindromic Repeats CRISPR-associated protein 9 (CRISPR-Cas9) system has revolutionized gene therapy. Plasmid vector delivery is the most commonly used method for integrating the CRISPR-Cas9 system into cells. However, such foreign cytosolic DNAs trigger an innate immune response (IIR) within cells, which can hinder gene editing by inhibiting transgene expression. Although some small molecules have been shown to avoid the action of IIR on plasmids, they only work on a single target and may also affect cell viability. A genetic approach that works at a comprehensive level for manipulating IIR is still lacking. Here, we designed and constructed several artificial nucleic acid molecules (ANAMs), which are combi

SUBMITTER: Zhan H 

PROVIDER: S-EPMC7533053 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature