Targeted Small Noncoding RNA-Directed Gene Activation in Human Cells

  • Caio Damski
  • Kevin V. MorrisEmail author
Part of the Methods in Molecular Biology book series (MIMB, volume 1173)


A growing body of evidence suggests that noncoding RNA (ncRNA) transcripts play a fundamental role in regulating gene expression via targeting epigenetic modifications to particular loci in the genome. Classical examples of such regulation are X-chromosome inactivation and genomic imprinting; however it is now clear that ncRNAs exert their influence over a wider array of genes throughout the metazoan genome. Accumulating evidence suggests that the ncRNAs act as guides for epigenetic silencing complexes to specific sites within the genome. Those ncRNAs involved in regulating the expression of particular protein-coding genes offer panoply of targets that when suppressed can result in derepression or activation of the ncRNA-targeted locus. Recent work has determined the underlying mechanisms involved in ncRNA-targeted epigenetic regulation in a subset of genes. These findings have resulted in a paradigm shift whereby targeted gene activation can be achieved, by targeting endogenous regulatory ncRNAs, producing potential novel treatments for genetic and infectious diseases where increases in gene expression are required.


Long noncoding RNA Antisense transcripts Small noncoding RNAs X chromosome inactivation Genomic imprinting Gene silencing Gene activation DNA methylation Histone modification 



The project was supported by NIHLB R01AI084406, NIAID R56 AI096861-01, and PO1 AI099783-01 to KVM.


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Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  1. 1.Biotechnology and Biomedical SciencesThe University of New South WalesSydneyAustralia
  2. 2.Molecular and Experimental MedicineThe Scripps Research InstituteLa JollaUSA

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