英国的国民保健制度开始为乙型地中海贫血等严重血液疾病提供Cashevy基因编辑治疗。 UK's NHS begins providing Casgevy gene-editing therapy for severe blood disorders like beta-thalassaemia.
联合王国国家保健服务处将开始向患有严重血病(如乙型地中海贫血)的病人提供首次基因编辑疗法,该疗法由Vertex制药公司和Crispr治疗公司开发。 The UK's National Health Service will begin providing the first gene-editing therapy, developed by Vertex Pharmaceuticals and Crispr Therapeutics, for patients with severe blood disorders such as beta-thalassaemia. Exagagongrogene autotemcel,又称Casgevy, 获准在没有骨髓移植的情况下用于严重乙型地中海贫血患者。 Exagamglogene autotemcel, also known as Casgevy, was approved for use in patients with severe beta-thalassaemia when no bone marrow transplant is available. 治疗方法是改变病人骨髓干细胞中的缺陷基因,使身体产生功能正常的血红蛋白,减少定期输血的需要,提高预期寿命。 The treatment works by modifying a faulty gene in the patient's bone marrow stem cells, allowing the body to produce functioning hemoglobin, reducing the need for regular blood transfusions, and improving life expectancy. Casgevy有可能为某些依赖输血的乙型地中海贫血患者提供潜在的治疗,使他们摆脱需要定期输血的负担和风险。 Casgevy holds the potential to represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions.