De-repressing LncRNA-targeted genes to upregulate gene expression: Focus on small molecule therapeutics

Roya Pedram Fatemi, Dmitry Velmeshev, Mohammad Ali Faghihi

Research output: Contribution to journalReview articlepeer-review

48 Scopus citations


Non-protein coding RNAs (ncRNAs) make up the overwhelming majority of transcripts in the genome and have recently gained attention for their complex regulatory role in cells, including the regulation of protein-coding genes. Furthermore, ncRNAs play an important role in normal development and their expression levels are dysregulated in several diseases. Recently, several long noncoding RNAs (lncRNAs) have been shown to alter the epigenetic status of genomic loci and suppress the expression of target genes. This review will present examples of such a mechanism and focus on the potential to target lncRNAs for achieving therapeutic gene upregulation by de-repressing genes that are epigenetically silenced in various diseases. Finally, the potential to target lncRNAs, through their interactions with epigenetic enzymes, using various tools, such as small molecules, viral vectors and antisense oligonucleotides, will be discussed. We suggest that small molecule modulators of a novel class of drug targets, lncRNA-protein interactions, have great potential to treat some cancers, cardiovascular disease, and neurological disorders.

Original languageEnglish (US)
Pages (from-to)e196
JournalMolecular Therapy - Nucleic Acids
StatePublished - 2014


  • Cancer
  • Cardiovascular disease
  • Epigenetics
  • Gene upregulation
  • Long intervening RNAs
  • Long noncoding RNA
  • Natural antisense transcript
  • Neurological disorders
  • Protein-RNA interactions
  • RNA structure

ASJC Scopus subject areas

  • Molecular Medicine
  • Drug Discovery


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