干细胞衍生的心脏细胞有望治疗先天性心脏病

由威斯康星大学麦迪逊分校医学和公共卫生学院的医学物理学教授Marina Emborg和明尼苏达州罗切斯特市梅奥诊所的内科科学家Timothy Nelson领导的一个研究小组最近在《细胞移植》杂志上报告说，由诱导多能干细胞培养的心肌细胞可以在压力过载的情况下融入猴子的心脏。

A research team led by Marina Emborg, professor of medical physics in the UW-Madison School of Medicine and Public Health, and Timothy Nelson, physician scientist at the Mayo Clinic in Rochester, Minnesota, reported recently in the journal Cell Transplantation that heart muscle cells grown from induced pluripotent stem cells can integrate into the hearts of monkeys with a state of pressure overload.

称为右心室功能障碍的压力过载经常影响先天性心脏缺陷的儿童。

Also referred to as right ventricular dysfunction, pressure overload often affects children with congenital heart defects.

患者会出现胸部不适、呼吸困难、心悸和身体肿胀，并可能导致心脏衰竭。

Patients experience chest discomfort, breathlessness, palpitations and body swelling, and can develop a weakened heart.

如果不及时治疗可能会致命。

The condition can be fatal if left untreated.

几乎所有的单心室先天性心脏缺陷，尤其是右心室的缺陷，最终都会导致心力衰竭。

Nearly all single ventricle congenital heart defects, particularly those in the right ventricle, eventually lead to heart failure.

手术是一个暂时的解决方案。最终，患者可能需要进行心脏移植。

Surgery to correct the defect is a temporary solution, according to the researchers. Eventually, patients may require a heart transplant.

研究人员将临床级人类诱导多能干细胞（从人类供体收集的细胞，诱导回到干细胞状态，然后发育成与心肌相容的细胞类型）移植到手术诱导右心室压力过载的恒河猴体内。

The researchers transplanted clinical-grade human induced pluripotent stem cells -- cells collected from human donors, coaxed back into a stem cell state and then developed into cell types compatible with heart muscle -- into rhesus macaque monkeys with surgically induced right ventricular pressure overload.

这些细胞成功地融入了周围宿主心肌（心脏的肌肉层）的组织中。

The cells successfully integrated into the organization of the surrounding host myocardium, the muscular layer of the heart.

作者注意到，在接受细胞移植的16只动物中，有5只发生了室性心动过速（心率增加），其中两只猴子出现了持续的心动过速。

The authors noted that episodes of ventricular tachycardia (an increased heart rate) occurred in five out of 16 animals receiving transplanted cells, with two monkeys presenting incessant tachycardia.

这些症状在19天内会消退。

These episodes resolved within 19 days.

该研究证实了将干细胞用于首个非人灵长类右心室压力过载模型的可行性和安全性。

The research proved the feasibility and safety of using stem cells in the first nonhuman primate model of right ventricular pressure overload.

在受损心脏中细胞成功整合和成熟是向先天性心脏缺陷临床应用迈出的有希望的一步。

The demonstration of successful integration and maturation of the cells into a compromised heart is a promising step towards the clinical application for congenital heart defects

Journal Reference:

Jodi Scholz, Frank J. Secreto, Joan Wobig, Joe Kurian, Clint Hagen, Alexandra Zinnen, Don Vu, Steven J. Johnson, Frank Cetta, Yasir Qureshi, Rachel Reams, Bryan Cannon, Christina M. Heyer, Minhwang Chang, Numrah Fadra, Jennifer Coonen, Heather A. Simmons, Andres Mejia, Jennifer M. Hayes, Puja Basu, Saverio Capuano, Viktoriya Bondarenko, Jeanette M. Metzger, Timothy J. Nelson, Marina E. Emborg. Human Stem Cell–Derived Cardiomyocytes Integrate Into the Heart of Monkeys With Right Ventricular Pressure Overload. Cell Transplantation, 2024; 33 DOI: 10.1177/09636897241290367