In vitro synthesis of modified rna for cardiac gene therapy

Nishat Sultana, Mohammad Tofael Kabir Sharkar, Yoav Hadas, Elena Chepurko, Lior Zangi

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

6 Scopus citations

Abstract

Modified mRNA (modRNA) is a promising new gene therapy approach that has safely and effectively delivered genes into different tissues, including the heart. Current efforts to use DNA-based or viral gene therapy to induce cardiac regeneration postmyocardial infarction (MI) or in heart failure (HF) have encountered key challenges, e.g., genome integration and delayed and noncontrolled expression. By contrast, modRNA is a transient, safe, non-immunogenic, and controlled gene delivery method that is not integrated into the genome. For most therapeutic applications, especially in regenerative medicine, the ability to deliver genes to the heart transiently and with control is vital for achieving therapeutic effect. Additionally, modRNA synthesis is comparatively simple and inexpensive compared to other gene delivery methods (e.g., protein), though a simple, clear in vitro transcription (IVT) protocol for synthesizing modRNA is needed for it to be more widely used. Here, we describe a simple and improved step-by-step IVT protocol to synthesize modRNA for in vitro or in vivo applications.

Original languageEnglish
Title of host publicationMethods in Molecular Biology
PublisherHumana Press Inc.
Pages281-294
Number of pages14
DOIs
StatePublished - 2021

Publication series

NameMethods in Molecular Biology
Volume2158
ISSN (Print)1064-3745
ISSN (Electronic)1940-6029

Keywords

  • Gene therapy
  • Modified mRNA
  • Myocardial infarction
  • mRNA stability
  • mRNA translation

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