Sector of Medicine, Scuola Superiore Sant'Anna, Pisa, Italy.
Transl Res. 2010 Sep;156(3):130-5. doi: 10.1016/j.trsl.2010.06.009. Epub 2010 Jul 15.
During the past 30 years, hundreds of pharmacological agents have been developed for the treatment of heart failure; yet few of them ultimately have been tested in patients. Such a disconcerting debacle has spurred the search for non pharmacological therapies, including those based on cardiac delivery of transgenes and stem cells. Cardiac gene therapy preceded stem cell therapy by approximately 10 years; however, both of them already have known an initial phase of enormous enthusiasm followed by moderate-to-strong skepticism, not necessarily justified. The aim of the present review is to discuss succinctly some key aspects of these 2 biological therapies and to argue that, after a phase of disillusionment, gene therapy for the failing heart likely will have the chance to regain the stage. In fact, discoveries in stem cell biology might revitalize gene therapy and, vice versa, gene therapy might potentiate synergistically the regenerative capacity of stem cells.
在过去的 30 年中，已经开发出数百种用于治疗心力衰竭的药理学药物；然而，其中很少有药物最终在患者中进行了测试。这种令人不安的失败促使人们寻找非药理学疗法，包括基于心脏传递转基因和干细胞的疗法。心脏基因治疗比干细胞治疗早了大约 10 年；然而，它们都已经经历了一个最初的阶段，即巨大的热情之后是适度到强烈的怀疑，这种怀疑并不一定是合理的。本综述的目的是简要讨论这两种生物疗法的一些关键方面，并认为，在经历了一个幻想破灭的阶段之后，心力衰竭的基因治疗可能有机会重新获得舞台。事实上，干细胞生物学的发现可能会使基因治疗重获生机，反之，基因治疗也可能协同增强干细胞的再生能力。
