Couple pay >$800k for a gene-editing therapy for their daughter. She died.

情侣花费超80万美元为女儿进行基因编辑治疗，但女儿不幸去世

HN 322 分 · 184 条评论 · 作者 Shortness8 · 来源 www.science.org · HN 讨论

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【评论】共 205 条 · 按 HN 热度顺序 · 楼中楼缩进展示

1L @maxall4 · 2026-07-24 06:23

I'm honestly quite shocked that the physicians/scientists involved would choose to use an AAV for a brain-targeted gene therapy. There is just so much data demonstrating that these vectors are quite immunoreactive: most of the approved gene therapies based on AAVs carry black box labels for liver failure caused by an immune reaction to the viral capsid. Admittedly, AAVs are the most derisked vector for gene therapies, but infusing them directly into someone's brain and expecting nothing bad to happen is, in my view, crazy.

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