ncRNAs in Therapeutics: Challenges and Limitations in Nucleic Acid-Based Drug Delivery

Int J Mol Sci. 2021 Oct 27;22(21):11596. doi: 10.3390/ijms222111596.

Abstract

Non-coding RNAs (ncRNAs) are emerging therapeutic tools but there are barriers to their translation to clinical practice. Key issues concern the specificity of the targets, the delivery of the molecules, and their stability, while avoiding "on-target" and "off-target" side effects. In this "ncRNA in therapeutics" issue, we collect several studies of the differential expression of ncRNAs in cardiovascular diseases, bone metabolism-related disorders, neurology, and oncology, and their potential to be used as biomarkers or therapeutic targets. Moreover, we review recent advances in the use of antisense ncRNAs in targeted therapies with a particular emphasis on their basic biological mechanisms, their translational potential, and future trends.

Keywords: delivery vehicles; nanoparticles; nanotechnology; ncRNAs; oligonucleotide therapy.

Publication types

  • Review

MeSH terms

  • Animals
  • Biomarkers / metabolism
  • Cardiovascular Diseases / genetics
  • Cardiovascular Diseases / therapy
  • Drug Delivery Systems / methods
  • Humans
  • Nucleic Acids / genetics*
  • RNA, Untranslated / genetics*

Substances

  • Biomarkers
  • Nucleic Acids
  • RNA, Untranslated