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mRNA 疗法对心血管疾病的未来影响。

Future Impact of mRNA Therapy on Cardiovascular Diseases.

机构信息

Houston Methodist Research Institute, Houston, Texas, US.

出版信息

Methodist Debakey Cardiovasc J. 2022 Dec 6;18(5):64-73. doi: 10.14797/mdcvj.1169. eCollection 2022.

Abstract

The silver lining of the recent pandemic was that it accelerated the emergence of messenger ribonucleic acid (mRNA) therapeutics. The great promise of mRNA therapeutics was highlighted by the speed at which the vaccines were created, tested, and proven to be relatively safe and highly effective. There are a wide variety of mRNA therapeutics now under development, and dozens of these are in clinical trials. These therapeutics are generating a major paradigm shift in medical therapy, including the treatment of cardiovascular disease. Most of the cardiovascular mRNA therapies are still in preclinical development, although a phase 2a trial of mRNA therapy for myocardial ischemia has been completed with promising results. The application of mRNA therapies to cardiovascular diseases is virtually limitless, and ongoing work includes mRNA therapies for myocardial ischemia, heart failure, arrhythmias, hypercholesterolemia, and arterial occlusive diseases. In addition, mRNA may be used to enhance cell therapies. In the future, mRNA therapies for cardiovascular disease are destined to supplant some of our current biologics and pharmacotherapies and will be used to treat previously untreatable cardiovascular diseases. Furthermore, mRNA therapies can be personalized, and they can be rapidly generated in current Good Manufacturing Practice facilities with a modest footprint, facilitating the rise of hospital-based regional centers of RNA therapeutics.

摘要

最近的大流行病带来的一线希望是,它加速了信使核糖核酸(mRNA)疗法的出现。mRNA 疗法的巨大前景体现在疫苗的研发、测试和证明相对安全和高度有效的速度上。现在有许多种 mRNA 疗法正在开发中,其中数十种正在进行临床试验。这些疗法正在引发医学治疗的重大范式转变,包括心血管疾病的治疗。尽管针对心肌缺血的 mRNA 疗法的 2a 期临床试验已经完成,结果令人鼓舞,但大多数心血管 mRNA 疗法仍处于临床前开发阶段。mRNA 疗法在心血管疾病中的应用几乎是无限的,目前正在进行的工作包括用于心肌缺血、心力衰竭、心律失常、高胆固醇血症和动脉阻塞性疾病的 mRNA 疗法。此外,mRNA 可用于增强细胞疗法。未来,心血管疾病的 mRNA 疗法注定要取代我们目前的一些生物制剂和药物疗法,并将用于治疗以前无法治疗的心血管疾病。此外,mRNA 疗法可以个性化,并且可以在具有适度占地面积的现行良好生产规范设施中快速生成,促进基于医院的 RNA 治疗区域中心的兴起。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6457/9733127/03e1d0ec187c/mdcvj-18-5-1169-g1.jpg

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