Investigating Friedreich Ataxia cardiomyopathy and ophthalmopathy using induced pluripotent stem cells

弗里德赖希共济失调（FRDA）没有有效的治疗方法。通常被诊断为儿童的病人最终会坐在轮椅上；许多人在运动、语言方面有困难，还会出现眼疾和糖尿病。大多数人在年轻时死于心力衰竭。在这个项目中，我们将研究来自FRDA患者的干细胞。在实验室里，这些干细胞将被转化为心脏和眼睛细胞，以研究FRDA患者出现症状的原因，并帮助开发新的药物来提供有效的治疗。

There is no effective treatment for Friedreich ataxia (FRDA). Patients, usually diagnosed as children, will end up in wheelchairs; many have difficulty with movement, speech, develop eye problems and diabetes. Most die at a young age from heart failure. In this project we will study stem cells from patients with FRDA. In the laboratory, these stem cells will be turned into heart and eye cells to study why patients with FRDA develop symptoms and to help develop new drugs to provide effective treatment.