Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy

Molecules. 2020 Jun 22;25(12):2866. doi: 10.3390/molecules25122866.

Abstract

The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.

Keywords: DNA; gene therapy; lipid nanoparticles; mRNA; nanoparticles; nucleic acids.

Publication types

  • Review

MeSH terms

  • DNA / genetics
  • DNA / therapeutic use
  • Gene Transfer Techniques*
  • Genetic Therapy / trends
  • Genetic Vectors / genetics*
  • Genetic Vectors / therapeutic use
  • Humans
  • Lipids / genetics*
  • Lipids / therapeutic use
  • Nanoparticles / chemistry
  • Polymers / chemistry*
  • Polymers / therapeutic use
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / therapeutic use

Substances

  • Lipids
  • Polymers
  • RNA, Small Interfering
  • DNA