Published online May 20, 2016. doi: 10.5493/wjem.v6.i2.37
Peer-review started: August 31, 2015
First decision: October 27, 2015
Revised: March 2, 2016
Accepted: March 17, 2016
Article in press: March 18, 2016
Published online: May 20, 2016
Core tip: Post-transcriptional microRNA-induced suppression of gene expression is a simple new, highly efficient technology to restrict transgene expression to a specific tissue. It is based on the insertion of a target sequence for a cell-specifically expressed microRNA, typically into the 3’ untranslated region of a transgene expression cassette. MicroRNA-induced regulation can result in an up to 100-fold reduction of transgene expression in tissues where expression is not desired. This targeting strategy can be used in combination with other targeting strategies to further improve vector specificity for gene therapeutic approaches.