在93%的试验病人中,CRISPR疗法治愈了镰状细胞病和乙型地中海贫血,没有重大安全问题。 A CRISPR therapy cured sickle cell disease and beta thalassemia in 93% of trial patients, with no major safety issues.

CRISPR 治疗学报告称,93%的病人在临床试验中接受了基于CRISPR的CTX0E03临床治疗,他们实现了镰状细胞疾病和乙型地中海贫血的输血独立,大多数症状减少,没有与基因编辑有关的严重安全问题。 CRISPR Therapeutics reported that 93% of patients in a clinical trial for its CRISPR-based therapy CTX0E03 achieved transfusion independence for sickle cell disease and beta thalassemia, with most experiencing reduced symptoms and no serious safety issues linked to gene editing. 这种治疗恢复了胎儿血红蛋白的活力,显示出有希望作为一种潜在的治疗方法,激发投资者的兴趣,并表明今后可能批准监管。 The treatment, which reactivates fetal hemoglobin, shows promise as a potential cure, boosting investor interest and signaling possible future regulatory approval. 在制造业的可扩展性和长期数据方面仍然存在挑战,但与Vertex制药公司的持续合作继续推进发展。 Challenges remain in manufacturing scalability and long-term data, but ongoing collaboration with Vertex Pharmaceuticals continues to advance development.