RNA Therapeutics in Cardiovascular Precision Medicine

Frontiers in Physiology
Ageliki LainaKonstantinos Stellos

Abstract

Since our knowledge on structure and function of messenger RNA (mRNA) has expanded from merely being an intermediate molecule between DNA and proteins to the notion that RNA is a dynamic gene regulator that can be modified and edited, RNA has become a focus of interest into developing novel therapeutic schemes. Therapeutic modulation of RNA molecules by DNA- and RNA-based therapies has broadened the scope of therapeutic targets in infectious diseases, cancer, neurodegenerative diseases and most recently in cardiovascular diseases as well. Currently, antisense oligonucleotides (ASO), small interfering RNAs (siRNAs), and microRNAs are the most widely applied therapeutic strategies to target RNA molecules and regulate gene expression and protein production. However, a number of barriers have to be overcome including instability, inadequate binding affinity and delivery to the tissues, immunogenicity, and off-target toxicity in order for these agents to evolve into efficient drugs. As cardiovascular diseases remain the leading cause of mortality worldwide, a large number of clinical trials are under development investigating the safety and efficacy of RNA therapeutics in clinical conditions such as familial hypercholesterolemia, di...Continue Reading

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Methods Mentioned

BETA
antisense oligonucleotides
chemical modifications
SELEX
antisense oligonucleotide
Exome Sequencing
ELISA

Clinical Trials Mentioned

NCT01200420
NCT01437059
NCT02314442
NCT01350960
NCT02414594
NCT02709850
NCT00742612
NCT00507338
NCT00808964
NCT01529424

Software Mentioned

Volanosersen

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