遗传增强
翻译(生物学)
医学
病毒学
生物
计算生物学
基因
遗传学
信使核糖核酸
作者
Thierry VandenDriessche,Marinee Chuah
标识
DOI:10.1016/j.ymthe.2022.11.006
摘要
Successful gene therapy for hemophilia A had been considered as one of the “holy grails” in the gene therapy field. An important milestone toward realizing this goal had been achieved (https://www.ema.europa.eu/en/news/first-gene-therapy-treat-severe-haemophilia) following the first conditional marketing authorization approval in the European Union for an adeno-associated virus type 5 (AAV5)-based gene therapy strategy.1 Most of the treated patients remained off prophylaxis and bleed-free 2 years after dosing and up to 5 years in a supporting trial.
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