CRISPR/Cas9: Principle, Applications, and Delivery through Extracellular Vesicles.

ABSTRACT: The establishment of CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) technology for eukaryotic gene editing opened up new avenues not only for the analysis of gene function but also for therapeutic interventions. While the original methodology allowed for targeted gene disruption, recent technological advancements yielded a rich assortment of tools to modify genes and gene expression in various ways. Currently, clinical applications of this technology fell short of expectations mainly due to problems with the efficient and safe delivery of CRISPR/Cas9 components to living organisms. The targeted in vivo delivery of therapeutic nucleic acids and proteins remain technically challenging and further limitations emerge, for instance, by unwant

SUBMITTER: Horodecka K 

PROVIDER: S-EPMC8200053 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature