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Updated: May 16, 2025
Delivery of Modified mRNA in a Myocardial Infarction Mouse Model
Published on: June 11, 2020
The Potential of RNA Therapeutics in Treating Cardiovascular Disease
Gayatri Mainkar1,2,3,4, Matteo Ghiringhelli1,2,3, Lior Zangi5,6,7
1Icahn School of Medicine at Mount Sinai, Cardiovascular Research Institute, New York, NY, 10029, USA.
Insights
RNA therapeutics offer new hope for cardiovascular diseases (CVDs) by targeting root causes, not just symptoms. This review explores RNA-based treatments for diverse CVD pathologies and their delivery strategies.
Area of Science:
- Cardiology
- Molecular Biology
- Genetics
Background:
- Cardiovascular diseases (CVDs) remain a leading global health burden despite medical advances.
- Current treatments often manage symptoms rather than addressing underlying causes.
- Novel therapeutic strategies are crucial for tackling CVDs effectively.
Purpose of the Study:
- To review the current landscape of RNA-based therapeutics for cardiovascular diseases (CVDs).
- To discuss classifications, mechanisms of action, and delivery strategies for RNA therapeutics in CVDs.
- To highlight potential CVD targets amenable to RNA-based interventions.
Main Methods:
- Literature review of RNA-based therapeutic strategies for CVDs.
- Analysis of different RNA molecule types and their functions in gene expression regulation.
- Examination of advancements in delivery systems for RNA therapeutics.
- Identification of specific CVD pathologies and targets for RNA-based treatments.
Main Results:
- RNA therapeutics can address diverse CVD mechanisms, including revascularization, regeneration, apoptosis prevention, lipid reduction, blood pressure control, fibrosis reversal, and genetic correction.
- Various RNA modalities show promise for treating a spectrum of cardiovascular conditions.
- Delivery strategies are critical for the clinical implementation of RNA-based CVD therapies.
Conclusions:
- RNA-based therapeutics represent a promising frontier for treating cardiovascular diseases by targeting fundamental causal pathways.
- The versatility of RNA molecules allows for tailored interventions across various CVD contexts.
- Further research and development in delivery technologies are essential for realizing the full potential of RNA therapeutics in cardiology.
Abstract:
Despite significant advances in cardiology over the past few decades, cardiovascular diseases (CVDs) remain the leading cause of global mortality and morbidity. This underscores the need for novel therapeutic interventions that go beyond symptom management to address the underlying causal mechanisms of CVDs. RNA-based therapeutics represent a new class of drugs capable of regulating specific genetic and molecular pathways, positioning them as strong candidates for targeting the root causes of a wide range of diseases. Moreover, owing to the vast diversity in RNA form and function, these molecules can be utilized to induce changes at different levels of gene expression regulation, making them suitable for a broad array of medical applications, even within a single disease context. Several RNA-based therapies are currently being investigated for their potential to address various CVD pathologies. These include treatments aimed at promoting cardiac revascularization and regeneration, preventing cardiomyocyte apoptosis, reducing harmful circulating cholesterols and fats, lowering blood pressure, reversing cardiac fibrosis and remodeling, and correcting the genetic basis of inherited CVDs. In this review, we discuss the current landscape of RNA therapeutics for CVDs, with an emphasis on their classifications, modes of action, advancements in delivery strategies and considerations for their implementation, as well as CVD targets with proven therapeutic potential.
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