Rett syndrome (RTT) is a neurodevelopmental disorder affecting mostly girls. A seemingly normal initial development is followed by developmental stagnation and regression, leading to severe mental impairment with autistic features, motor dysfunction, irregular breathing and epilepsy. Currently, a cure does not exist. Due to the close association of RTT with mitochondrial alterations, cellular redox-impairment and oxidative stress, compounds stabilizing mitochondrial function, cellular redox-homeostasis, and oxidant detoxification are increasingly considered as treatment concepts. Indeed, antioxidants and free-radical scavengers ameliorate certain aspects of the complex and severe clinical presentation of RTT. To further evaluate these strategies, reliable biosensors are needed to quantify redox-conditions in brain and peripheral organs of mouse models or in patient-derived cells. Genetically-encoded redox-sensors meet these requirements. Expressed in transgenic mouse-models such as our unique Rett-redox indicator mice, they will report for any cell type desired the severity of oxidant stress throughout the various disease stages of RTT. Furthermore, these sensors will be crucial to evaluate in vitro and in vivo the outcome of mitochondria- and redox-balance targeted treatments.

Rett综合征（RTT）是一种神经发育障碍，主要影响女孩。看似正常的初始发育之后是发育停滞和消退，导致严重的精神障碍，包括自闭症，运动功能障碍，呼吸不规则和癫痫病。当前，不存在治愈方法。由于RTT与线粒体改变，细胞氧化还原损伤和氧化应激密切相关，稳定线粒体功能，细胞氧化还原稳态和氧化剂解毒的化合物越来越多地被视为治疗概念。实际上，抗氧化剂和自由基清除剂改善了RTT复杂而严重的临床表现的某些方面。为了进一步评估这些策略，需要可靠的生物传感器来量化小鼠模型的大脑和周围器官或患者来源的细胞中的氧化还原条件。经过基因编码的氧化还原传感器可以满足这些要求。在转基因小鼠模型中表达，例如我们独特的Rett-redox指示剂小鼠，它们将报告在RTT各个疾病阶段中所需的任何细胞类型氧化应激的严重程度。此外，这些传感器对于评估体外和体内线粒体和氧化还原平衡靶向治疗的结果。