TY - JOUR
T1 - RNA-based therapeutics in cardiovascular disease
AU - Pham, Tan Phát
AU - Kremer, Veerle
AU - Boon, Reinier A.
PY - 2020
Y1 - 2020
N2 - Purpose of reviewCardiovascular disease is the leading cause of death globally, with the number of deaths rising every year. Much effort has gone into development of new treatment strategies. Many RNA species have important regulatory functions in disease initiation and progression, providing interesting new treatment options. This review focuses on different classes of RNA-based therapeutics and provides examples of current clinical and preclinical studies. Current challenges that prevent clinical translation and possibilities to overcome them will be discussed.Recent findingsDifferent RNA-based molecules have been developed, such as antisense oligos, microRNA mimics and small interfering RNAs. Modifications are used to prevent degradation and immune activation and improve affinity. Additionally, in order to improve delivery of the RNA molecules to the target tissues, viral or nonviral vectors can be used.SummaryRNA-based therapy has been shown to be a promising new treatment strategy for different disorders. However, several challenges, such as delivery problems and low efficacy remain. Future research will likely focus on effective delivery to target tissues in order to improve efficacy and avoid harmful side-effects.
AB - Purpose of reviewCardiovascular disease is the leading cause of death globally, with the number of deaths rising every year. Much effort has gone into development of new treatment strategies. Many RNA species have important regulatory functions in disease initiation and progression, providing interesting new treatment options. This review focuses on different classes of RNA-based therapeutics and provides examples of current clinical and preclinical studies. Current challenges that prevent clinical translation and possibilities to overcome them will be discussed.Recent findingsDifferent RNA-based molecules have been developed, such as antisense oligos, microRNA mimics and small interfering RNAs. Modifications are used to prevent degradation and immune activation and improve affinity. Additionally, in order to improve delivery of the RNA molecules to the target tissues, viral or nonviral vectors can be used.SummaryRNA-based therapy has been shown to be a promising new treatment strategy for different disorders. However, several challenges, such as delivery problems and low efficacy remain. Future research will likely focus on effective delivery to target tissues in order to improve efficacy and avoid harmful side-effects.
KW - RNA therapeutics
KW - cardiovascular
KW - clinical trial
KW - delivery
KW - nanoparticle
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85083041891&origin=inward
UR - https://www.ncbi.nlm.nih.gov/pubmed/32068614
UR - http://www.scopus.com/inward/record.url?scp=85083041891&partnerID=8YFLogxK
U2 - https://doi.org/10.1097/HCO.0000000000000724
DO - https://doi.org/10.1097/HCO.0000000000000724
M3 - Review article
C2 - 32068614
SN - 0268-4705
VL - 35
SP - 191
EP - 198
JO - Current opinion in cardiology
JF - Current opinion in cardiology
IS - 3
ER -