A critical analysis of codon optimization in human therapeutics

Trends Mol Med. 2014 Nov;20(11):604-13. doi: 10.1016/j.molmed.2014.09.003. Epub 2014 Sep 25.

Abstract

Codon optimization describes gene engineering approaches that use synonymous codon changes to increase protein production. Applications for codon optimization include recombinant protein drugs and nucleic acid therapies, including gene therapy, mRNA therapy, and DNA/RNA vaccines. However, recent reports indicate that codon optimization can affect protein conformation and function, increase immunogenicity, and reduce efficacy. We critically review this subject, identifying additional potential hazards including some unique to nucleic acid therapies. This analysis highlights the evolved complexity of codon usage and challenges the scientific bases for codon optimization. Consequently, codon optimization may not provide the optimal strategy for increasing protein production and may decrease the safety and efficacy of biotech therapeutics. We suggest that the use of this approach is reconsidered, particularly for in vivo applications.

Keywords: A-to-I editing; codon optimization; gene therapy; mRNA therapy; tRNA wobble; vaccine.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Codon*
  • Gene Expression Regulation
  • Genetic Engineering* / methods
  • Genetic Therapy*
  • Humans
  • Peptides / genetics
  • Peptides / metabolism
  • Peptides / therapeutic use
  • Protein Engineering
  • RNA Editing
  • RNA, Messenger / genetics
  • RNA, Transfer / chemistry
  • Recombinant Proteins / genetics
  • Recombinant Proteins / metabolism
  • Recombinant Proteins / therapeutic use*

Substances

  • Codon
  • Peptides
  • RNA, Messenger
  • Recombinant Proteins
  • RNA, Transfer