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利用RNA疗法治疗缺血性心力衰竭:TRAIN-HEART研究历程

Harnessing the power of RNA therapeutics in treating ischemic heart failure: the TRAIN-HEART story.

作者信息

Albert Alba M, Balamurali Deepak, Beslika Evangelia, Fernandez Ileana D, Genedy Hussein H, Babaei Khorzoughi Reyhaneh, Lauta Francesca Cecilia, Peppas Panagiotis, Ragone Isabella, Rizzari Giorgia, Sansonetti Marida, Silva Joana, Spanò Giulia, Wrona Kinga M

机构信息

Cardiac Research, Regenerative Therapies, Target Discovery & Imaging, Miltenyi Biotec B.V. & Co. KG, Bergisch Gladbach, Germany.

Institute for Molecular and Translational Therapeutic Strategies (IMTTS), Hannover Medical School, Hannover, Germany.

出版信息

Front Cardiovasc Med. 2024 Jan 11;10:1228160. doi: 10.3389/fcvm.2023.1228160. eCollection 2023.

Abstract

Ischemic heart disease (IHD) is one of the world's foremost killers, accounting for 16% of all deaths worldwide. IHD is the main cause of heart failure (HF), as it leads to pathological changes in the heart, improper pumping function and eventual death. Therapeutic interventions usually follow a systemic general strategy for all heart failure subtypes due to the lack of a deep understanding of the disease mechanisms. Hence, HF and IHD therapeutics need groundbreaking concepts to guide the development of a new therapeutics class that tackles the disease at a molecular level. The TRAIN-HEART consortium, a Marie Skłodowska-Curie Actions Innovative Training Network (MSCA-ITN) funded by the European Commission, was established with the goal of filling that gap and developing RNA-based cardiovascular therapeutics. Created in the context of the Horizon 2020 research and innovation program, TRAIN-HEART comprises three key work packages (WPs) focusing on the pathogenesis of heart disease (WP1), the therapeutic potential of RNA therapeutics (WP2), and the development of new efficient delivery systems (WP3). Fifteen international early stage researchers (ESRs) from multiple complementary scientific disciplines were recruited to collaborate with a network of PIs from nine academic and eight non-academic partners in various disciplines to fully harness their collective potential for the betterment of HF treatment. This article provides an overview of the benefits of being part of an MSCA-ITN, with its different training and networking opportunities, maximizing ESRs' potential and broadening collaborative research possibilities. Finally, it describes what was like to do a PhD during the COVID-19 pandemic, with all the uncertainty and concern attached to it. Luckily, TRAIN-HEART stood out as a proactive network, finding new initiatives and alternatives to promote scientific and personal development. By bringing together leading academic teams, (biotech) companies, and highly motivated researchers, TRAIN-HEART is expanding scientific horizons and accelerating future development of effective RNA-based therapies to treat IHD.

摘要

缺血性心脏病(IHD)是全球主要杀手之一,占全球所有死亡人数的16%。IHD是心力衰竭(HF)的主要原因,因为它会导致心脏发生病理变化、泵血功能异常并最终导致死亡。由于对疾病机制缺乏深入了解,治疗干预通常遵循针对所有心力衰竭亚型的系统性总体策略。因此,HF和IHD治疗需要开创性的概念来指导开发一类新的分子水平治疗疾病的疗法。由欧盟委员会资助的玛丽·居里行动创新培训网络(MSCA-ITN)——TRAIN-HEART联盟的成立,旨在填补这一空白并开发基于RNA的心血管疗法。TRAIN-HEART是在“地平线2020”研究与创新计划背景下创建的,包括三个关键工作包(WP),分别聚焦于心脏病发病机制(WP1)、RNA疗法的治疗潜力(WP2)以及新型高效递送系统的开发(WP3)。来自多个互补科学学科的15名国际早期研究人员(ESR)被招募,与来自九个学术和八个非学术合作伙伴的不同学科的PI网络合作,以充分发挥他们的集体潜力,改善HF治疗。本文概述了成为MSCA-ITN一员的好处,包括其不同的培训和网络机会,最大限度地发挥ESR的潜力并拓宽合作研究可能性。最后,它描述了在新冠疫情期间攻读博士学位的情况,以及随之而来的所有不确定性和担忧。幸运的是,TRAIN-HEART作为一个积极主动的网络脱颖而出,找到了促进科学和个人发展的新举措和替代方案。通过汇聚顶尖学术团队、(生物技术)公司和积极性高的研究人员,TRAIN-HEART正在拓展科学视野,加速基于RNA的有效疗法治疗IHD的未来发展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2f81/10809146/daa681aab933/fcvm-10-1228160-g001.jpg

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