Synthetic long non-coding RNAs [SINEUPs] rescue defective gene expression in vivo.

ABSTRACT: Non-coding RNAs provide additional regulatory layers to gene expression as well as the potential to being exploited as therapeutic tools. Non-coding RNA-based therapeutic approaches have been attempted in dominant diseases, however their use for treatment of genetic diseases caused by insufficient gene dosage is currently more challenging. SINEUPs are long antisense non-coding RNAs that up-regulate translation in mammalian cells in a gene-specific manner, although, so far evidence of SINEUP efficacy has only been demonstrated in in vitro systems. We now show that synthetic SINEUPs effectively and specifically increase protein levels of a gene of interest in vivo. We demonstrated that SINEUPs rescue haploinsufficient gene dosage in a medakafish model of a human disorder leading to ameliorat

SUBMITTER: Indrieri A 

PROVIDER: S-EPMC4893607 | biostudies-literature | 2016 Jun

REPOSITORIES: biostudies-literature