疾病
医学
基因
遗传增强
细胞
生物
遗传学
内科学
标识
DOI:10.58347/tml.2024.1694a
摘要
Two cell-based gene therapies – exagamglogene autotemcel (Casgevy – Vertex) and lovotibeglogene autotemcel (Lyfgenia – Bluebird Bio) – have been approved by the FDA for treatment of sickle cell disease in patients ≥12 years old with recurrent vaso-occlusive crises. They are the first gene therapies to be approved in the US for use in sickle cell disease; Casgevy is the first treatment to be approved in the US that uses CRISPR/Cas9 gene-editing technology.
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