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全球首例!婴儿接受定制化基因编辑成功治疗罕见病

一个国际研究团队报告了全球首例为人类患者定制的基因编辑疗法。一名 9 个月大的婴儿患有致命的氨甲酰磷酸合成酶 1 (CPS1) 缺乏症,研究人员利用 CRISPR 基因编辑技术,在 6 个月内为其开发并实施了定制化治疗,成功修复了婴儿肝细胞中的致病基因突变。截至今年 4 月,该婴儿接受了 3 剂基因编辑治疗,未出现严重不良反应,血液中的有毒氨积聚得到逆转。美国国立卫生研究院称,这是基因疗法首次在人类患者中实现定制化临床应用,为未来开发针对其他罕见病的精准疗法奠定了基础。

这项突破性的治疗被称为“kayjayguran abengcemeran”(简称 k-abe),通过脂质纳米颗粒将新一代碱基编辑系统输送到患者肝细胞,直接修改致病基因。治疗后监测显示,患儿血氨浓度等指标已恢复正常,健康状况持续改善。专家认为,碱基编辑技术的应用是此次成功的关键。尽管长期疗效和安全性仍需进一步观察,但这项成果标志着基因编辑在罕见病治疗领域迈出了重要一步。

The New England Journal of Medicine

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全球首例!婴儿接受定制化基因编辑成功治疗罕见病

一个国际研究团队报告了全球首例为人类患者定制的基因编辑疗法。一名 9 个月大的婴儿患有致命的氨甲酰磷酸合成酶 1 (CPS1) 缺乏症,研究人员利用 CRISPR 基因编辑技术,在 6 个月内为其开发并实施了定制化治疗,成功修复了婴儿肝细胞中的致病基因突变。截至今年 4 月,该婴儿接受了 3 剂基因编辑治疗,未出现严重不良反应,血液中的有毒氨积聚得到逆转。美国国立卫生研究院称,这是基因疗法首次在人类患者中实现定制化临床应用,为未来开发针对其他罕见病的精准疗法奠定了基础。

这项突破性的治疗被称为“kayjayguran abengcemeran”(简称 k-abe),通过脂质纳米颗粒将新一代碱基编辑系统输送到患者肝细胞,直接修改致病基因。治疗后监测显示,患儿血氨浓度等指标已恢复正常,健康状况持续改善。专家认为,碱基编辑技术的应用是此次成功的关键。尽管长期疗效和安全性仍需进一步观察,但这项成果标志着基因编辑在罕见病治疗领域迈出了重要一步。

The New England Journal of Medicine

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A leaked Telegram discussion by 50 so-called crypto influencers has exposed the extraordinary steps they take in order to profit on the back off unsuspecting defi investors. According to a leaked screenshot of the chat, an elaborate plan to defraud defi investors using the worthless “$Few” tokens had been hatched. $Few tokens would be airdropped to some of the influencers who in turn promoted these to unsuspecting followers on Twitter.

Telegram today rolling out an update which brings with it several new features.The update also adds interactive emoji. When you send one of the select animated emoji in chat, you can now tap on it to initiate a full screen animation. The update also adds interactive emoji. When you send one of the select animated emoji in chat, you can now tap on it to initiate a full screen animation. This is then visible to you or anyone else who's also present in chat at the moment. The animations are also accompanied by vibrations. This is then visible to you or anyone else who's also present in chat at the moment. The animations are also accompanied by vibrations.

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