Modified mRNA as an alternative to plasmid DNA (pDNA) for transcript replacement and vaccination therapy

Hyewon Youn, June Key Chung

Research output: Contribution to journalReview article

27 Citations (Scopus)

Abstract

Introduction: Current gene therapy involves replacement of defective gene by delivery of healthy genetic material to precede normal function. Virus-mediated gene delivery is the most successful and efficient method for gene therapy, but it has been challenged due to serious safety concerns. Conversely, gene delivery using plasmid DNA (pDNA) is considered safer, but its transfection efficiency is much lower than virus-mediated gene transfer. Recently, mRNA has been suggested as an alternative option to avoid undesired insertion of delivered DNA sequences with higher transfection efficiency and stability.Area covered: In this review, we summarize the currently available strategies of mRNA modification to increase the therapeutic efficacy; we also highlight the recent improvements of mRNA delivery for in vivo applications of gene therapy.Expert opinion: The use of mRNA-based gene transfer could indeed be a promising new strategy for gene therapy. Notable advantages include no risk of integration into the genomic DNA, adjustable gene expression and easier modulation of the immune system. By reducing or utilizing the immunogenic properties, mRNA offers a promising tool for gene/or transcript replacement.

Original languageEnglish
Pages (from-to)1337-1348
Number of pages12
JournalExpert Opinion on Biological Therapy
Volume15
Issue number9
DOIs
StatePublished - 2 Sep 2015

Fingerprint

Gene therapy
Vaccination
Plasmids
Messenger RNA
Genes
DNA
Genetic Therapy
Gene transfer
Viruses
Therapeutics
Transfection
Immune system
DNA sequences
Gene expression
Expert Testimony
Modulation
Immune System
Safety
Gene Expression

Keywords

  • Chemical modification
  • delivery
  • gene therapy
  • mRNA

Cite this

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Modified mRNA as an alternative to plasmid DNA (pDNA) for transcript replacement and vaccination therapy. / Youn, Hyewon; Chung, June Key.

In: Expert Opinion on Biological Therapy, Vol. 15, No. 9, 02.09.2015, p. 1337-1348.

Research output: Contribution to journalReview article

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