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Synthetic long non-coding RNAs SINEUPs rescue defective gene expression in vivo
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Synthetic long non-coding RNAs SINEUPs rescue defective gene expression in vivo
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Journal Article
Synthetic long non-coding RNAs SINEUPs rescue defective gene expression in vivo
2016
Overview
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 amelioration of the disease phenotype. Our results demonstrate that SINEUPs act through mechanisms conserved among vertebrates and that SINEUP technology can be successfully applied
in vivo
as a new research and therapeutic tool for gene-specific up-regulation of endogenous functional proteins.
Publisher
Nature Publishing Group UK,Nature Publishing Group
Subject
/ 38/77
/ 38/90
/ 42/35
/ 45/41
/ 64/116
/ 82
/ 82/29
/ Animals
/ Biological Products - administration & dosage
/ Biological Products - metabolism
/ Cloning
/ Disease
/ Gene Expression Regulation - drug effects
/ Genetic Diseases, X-Linked - therapy
/ Genomes
/ Humanities and Social Sciences
/ Humans
/ Oryzias
/ Proteins
/ RNA, Long Noncoding - administration & dosage
/ RNA, Long Noncoding - genetics
/ RNA, Long Noncoding - metabolism
/ Science
