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Innovators want pills to treat sickle cell disease. Can they match gene therapy?
Nature Biotechnology ( IF 46.9 ) Pub Date : 2024-03-15 , DOI: 10.1038/s41587-024-02179-2
Cormac Sheridan

Although CRISPR-based gene therapy for sickle cell disease offers transformative outcomes, drugmakers are striving to develop treatments that are easy to manufacture and can reach much larger numbers of patients.

中文翻译:

创新者想要治疗镰状细胞病的药物。它们能与基因疗法相匹配吗?

尽管基于 CRISPR 的镰状细胞病基因疗法提供了变革性的结果,但制药商正在努力开发易于生产且可以惠及更多患者的治疗方法。
更新日期:2024-03-16
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