Engineering, delivery, and biological validation of artificial microRNA clusters for gene therapy applications.

ABSTRACT: The cellular machinery regulating microRNA biogenesis and maturation relies on a small number of simple steps and minimal biological requirements and is broadly conserved in all eukaryotic cells. The same holds true in disease. This allows for a substantial degree of freedom in the engineering of transgenes capable of simultaneously expressing multiple microRNAs of choice, allowing a more comprehensive modulation of microRNA landscapes, the study of their functional interaction, and the possibility of using such synergism for gene therapy applications. We have previously engineered a transgenic cluster of functionally associated microRNAs to express a module of suppressed microRNAs in brain cancer for therapeutic purposes. Here, we provide a detailed protocol for the design, cloning, deliv

SUBMITTER: Bhaskaran V 

PROVIDER: S-EPMC7089775 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature