King's College London, British Heart Foundation Centre of Research Excellence, School of Cardiovascular Medicine & Sciences, London, UK.
Department of Medical, Surgical and Health Sciences, University of Trieste and International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
Curr Opin Genet Dev. 2021 Oct;70:48-53. doi: 10.1016/j.gde.2021.05.007. Epub 2021 Jun 4.
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.
对于心肌梗死和心力衰竭,人们迫切需要开发新的疗法。一种新颖而令人兴奋的治疗可能性是通过刺激心肌细胞的内在增殖能力来实现心脏再生。利用病毒载体,在小动物和大动物中均已获得了 miRNA 诱导心脏再生的概念验证证据。然而,更适用于临床的策略是开发用于 RNA 治疗药物的脂质介导的纳米技术,作为合成分子。最近,稳定核酸脂质颗粒 (SNALP) 平台在生成纳米尺寸、高效且无炎症的脂质纳米颗粒方面取得了成功,为通过心导管术开发 miRNA 的可注射纳米制剂铺平了道路。