Sickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD.
Blood. 2024 Mar 7;143(10):866-871. doi: 10.1182/blood.2023021167.
Pyruvate kinase (PK) is a key enzyme in glycolysis, the sole source of adenosine triphosphate, which is essential for all energy-dependent activities of red blood cells. Activating PK shows great potential for treating a broad range of hemolytic anemias beyond PK deficiency, because they also enhance activity of wild-type PK. Motivated by observations of sickle-cell complications in sickle-trait individuals with concomitant PK deficiency, activating endogenous PK offers a novel and promising approach for treating patients with sickle-cell disease.
丙酮酸激酶（PK）是糖酵解的关键酶，是三磷酸腺苷（ATP）的唯一来源，ATP 是所有红细胞依赖能量活动所必需的。激活 PK 除了对 PK 缺乏症以外的广泛溶血性贫血具有很大的治疗潜力，因为它还增强了野生型 PK 的活性。受镰状细胞特质个体中伴随 PK 缺乏症的镰状细胞并发症的观察结果的启发，激活内源性 PK 为治疗镰状细胞病患者提供了一种新颖且有前途的方法。
