Current Opinion in Genetics & Development ( IF 3.7 ) Pub Date : 2021-06-04 , DOI: 10.1016/j.gde.2021.05.007 Josef Huntington 1 , Manendra Pachauri 2 , Hashim Ali 1 , Mauro Giacca 3
There is an impelling need to develop new therapeutics for myocardial infarction and heart failure. A novel and exciting therapeutic possibility is to achieve cardiac regeneration through the stimulation of the endogenous capacity of cardiomyocytes to proliferate. Proof-of-concept evidence of microRNA-induced cardiac regeneration is available in both small and large animals using viral vectors. However, a clinically more applicable strategy is the development of lipid-mediated nanotechnologies for the administration of RNA therapeutics as synthetic molecules. The recent success of the Stable Nucleic Acid Lipid Particle (SNALP) platform for the generation of nanosized, efficient and non-inflammatory lipid nanoparticles paves the way to the development of injectable nanoformulations of microRNAs through cardiac catheterisation.
中文翻译:
用于心脏再生的 RNA 干扰疗法
迫切需要开发用于心肌梗塞和心力衰竭的新疗法。一种新颖且令人兴奋的治疗可能性是通过刺激心肌细胞的内源性增殖能力来实现心脏再生。使用病毒载体的小型和大型动物都可以获得 microRNA 诱导的心脏再生的概念验证证据。然而,临床上更适用的策略是开发脂质介导的纳米技术,以将 RNA 治疗剂作为合成分子给药。稳定核酸脂质颗粒 (SNALP) 平台最近取得的成功,用于生成纳米级、高效和非炎症性脂质纳米颗粒,这为通过心脏导管插入开发可注射的 microRNA 纳米制剂铺平了道路。