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Gene therapy for hemophilia A
Nature Medicine ( IF 82.9 ) Pub Date : 2021-11-30 , DOI: 10.1038/d41591-021-00072-y
Karen O’Leary

In a phase 1/2 trial, an investigational gene therapy brought about sustained expression of clotting factor VII and a dramatic reduction in the bleeding rate.

中文翻译:

血友病A的基因治疗

在 1/2 期试验中,一项研究性基因治疗使凝血因子 VII 持续表达并显着降低出血率。
更新日期:2021-11-30
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