Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities

Yale J Biol Med. 2017 Dec 19;90(4):583-598. eCollection 2017 Dec.

Abstract

Since their invention in 1991, peptide nucleic acids (PNAs) have been used in a myriad of chemical and biological assays. More recently, peptide nucleic acids have also been demonstrated to hold great potential as therapeutic agents because of their physiological stability, affinity for target nucleic acids, and versatility. While recent modifications in their design have further improved their potency, their preclinical development has reached new heights due to their combination with recent advancements in drug delivery. This review focuses on recent advances in PNA therapeutic applications, in which chemical modifications are made to improve PNA function and nanoparticles are used to enhance PNA delivery.

Keywords: Peptide Nucleic Acids; anti-miR therapy; anti-miRs; anti-sense; antigene; antisense therapy; gene editing; gene therapy; nanoparticles; oncomiRs.

Publication types

  • Review

MeSH terms

  • Antisense Elements (Genetics)
  • Drug Delivery Systems / methods*
  • Drug Stability
  • Gene Editing
  • Gene Expression Regulation / drug effects*
  • Humans
  • MicroRNAs
  • Molecular Structure
  • Nanoparticles / administration & dosage
  • Nanoparticles / chemistry
  • Peptide Nucleic Acids / administration & dosage*
  • Peptide Nucleic Acids / chemistry
  • Peptide Nucleic Acids / pharmacokinetics
  • Peptide Nucleic Acids / therapeutic use*
  • Solubility

Substances

  • Antisense Elements (Genetics)
  • MicroRNAs
  • Peptide Nucleic Acids