英国批准世界首例对严重镰状细胞疾病的CRISPR基因编辑疗法。 UK approves world's first CRISPR gene-editing treatment for severe sickle cell disease.

联合王国国家卫生局(NHS)批准了165万英镑的基因编辑疗法,即Casgevy(exa-cel),用于治疗患有严重镰状细胞疾病的病人。 The UK's National Health Service (NHS) has approved a £1.65 million gene-editing therapy, Casgevy (exa-cel), for patients with severe sickle cell disease. 治疗是首先使用基因编辑工具CRISPR的治疗,它编辑病人干细胞中的有缺陷的基因,以产生健康的红细胞。 The treatment, the first to use the gene-editing tool CRISPR, edits faulty genes in a patient's stem cells to produce healthy red blood cells. 每年为大约50名病人核准治疗,为潜在的治疗提供了希望,并将在伦敦、曼彻斯特和伯明翰的专家中心提供治疗。 Approved for around 50 patients annually, it offers hope for a potential cure and will be available at specialist centers in London, Manchester, and Birmingham. 这一突破可使联合王国约1,700名个人受益。 This breakthrough could benefit about 1,700 individuals in the UK.