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Sickle cell gene therapies approach watershed
Nature Biotechnology ( IF 46.9 ) Pub Date : 2021-11-09 , DOI: 10.1038/s41587-021-01121-0
Cormac Sheridan 1
Affiliation  

Early results promise a one-off treatment, but gene-editing and gene therapies will have to show durable protection and be affordable to the many patients with sickle cell disease in the world.

中文翻译:

镰状细胞基因疗法接近分水岭

早期结果承诺一次性治疗,但基因编辑和基因疗法必须显示出持久的保护作用,并且对世界上许多镰状细胞病患者来说是负担得起的。
更新日期:2021-11-10
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