Abstract

Modified mRNA (modRNA) is a safe and effective vector for gene-based therapies. Notably, the safety of modRNA has been validated through COVID-19 vaccines which incorporate modRNA technology to translate spike proteins. Alternative gene delivery methods using plasmids, lentiviruses, adenoviruses, and adeno-associated viruses have suffered from key challenges such as genome integration, delayed and uncontrolled expression, and immunogenic responses. However, modRNA poses no risk of genome integration, has transient and rapid expression, and lacks an immunogenic response. Our lab utilizes modRNA-based therapies to promote cardiac regeneration following myocardial infarction and heart failure. We have also developed and refined an optimized and economical method for synthesis of modRNA. Here, we provide an updated methodology with improved translational efficiency for in vitro and in vivo application.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages77-87
Number of pages11
DOIs
StatePublished - 2022

Publication series

NameMethods in Molecular Biology
Volume2573
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029

Keywords

  • Cardiac gene therapy
  • Gene therapy
  • Gene-based therapy
  • Genetically based therapy
  • In vitro transcription
  • Modified mRNA
  • Synthetic mRNA
  • mRNA translation

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