Related Experiment Video
Updated: Jul 10, 2026

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
Published on: March 29, 2019
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and
Brian D Brown1, Bernhard Gentner, Alessio Cantore
1San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, via Olgettina, 58, 20132 Milan, Italy.
Abstract:
We have shown previously that transgene expression can be suppressed in hematopoietic cells using vectors that are responsive to microRNA (miRNA) regulation. Here we investigate the potential of this approach for more sophisticated control of transgene expression. Analysis of the relationship between miRNA expression levels and target mRNA suppression suggested that suppression depends on a threshold miRNA concentration. Using this information, we generated vectors that rapidly adjust transgene expression in response to changes in miRNA expression. These vectors sharply segregated transgene expression between closely related states of therapeutically relevant cells, including dendritic cells, hematopoietic and embryonic stem cells, and their progeny, allowing positive/negative selection according to the cells' differentiation state. Moreover, two miRNA target sites were combined to restrict transgene expression to a specific cell type in the liver. Notably, the vectors did not detectably perturb endogenous miRNA expression or regulation of natural targets. The properties of miRNA-regulated vectors should allow for safer and more effective therapeutic applications.
More Related Videos
Related Concept Videos
MicroRNAs
MicroRNAs
MicroRNAs
Regulation of Expression at Multiple Steps
Regulation of Expression Occurs at Multiple Steps
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
Forced Transdifferentiation
Artificial transdifferentiation occurs...

