Developments in lncRNA drug discovery: where are we heading?

Ilya Blokhin, Olga Khorkova, Jane Hsiao, Claes Wahlestedt

Research output: Contribution to journalReview articlepeer-review

24 Scopus citations


Introduction: The central dogma of molecular biology, which states that the only role of long RNA transcripts is to convey information from gene to protein, was brought into question in recent years due to discovery of the extensive presence and complex roles of long noncoding RNAs (lncRNAs). Furthermore, lncRNAs were found to be involved in pathogenesis of multiple diseases and thus represent a new class of therapeutic targets. Translational efforts in the lncRNA field have been augmented by progress in optimizing the chemistry and delivery platforms of lncRNA-targeting modalities, including oligonucleotide-based drugs and CRISPR-Cas9. Areas covered: This review covers the current advances in characterizing diversity and biological functions of lncRNA focusing on their therapeutic potential in selected therapeutic areas. Expert opinion: Due to accelerating parallel progress in lncRNA biology and lncRNA-compatible therapeutic modalities, it is likely that lncRNA-dependent mechanisms of pathogenesis will soon be targeted in various disorders, including neurological, psychiatric, cardiovascular, infectious diseases, and cancer. Significant efforts, however, are still required to better understand the biology of both lncRNAs and lncRNA-targeting drugs. Further work is needed in the areas of lncRNA nomenclature, database representation, intra/interfield communication, and education of the community at large.

Original languageEnglish (US)
Pages (from-to)837-849
Number of pages13
JournalExpert Opinion on Drug Discovery
Issue number9
StatePublished - Sep 2 2018


  • CRISPR-Cas9
  • Cancer
  • lncRNA
  • neurological
  • oligonucleotides
  • psychiatric

ASJC Scopus subject areas

  • Drug Discovery


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