Carl Juno团队发表在Science上的新研究表明,CRISPR基因编辑后的T细胞在癌症患者中可存活数月

2020-02-11 不详 MedSci原创

发表在Science杂志上的1期临床试验结果,证明CRISPR-Cas9基因编辑后的T细胞在患者体内可以保留几个月。并且分离接受治疗数月后患者体内基因编辑后的T细胞,在体外进行实验时仍然可以杀死癌症。

发表在Science杂志上的1期临床试验结果,证明CRISPR-Cas9基因编辑后的T细胞在患者体内可以保留几个月。并且分离接受治疗数月后患者体内基因编辑后的T细胞,在体外进行实验时仍然可以杀死癌症。

嵌合抗原受体(CAR)-T疗法是提取患者自身的T细胞,在体外进行修饰后再回输到患者体内发挥抗肿瘤作用。CAR-T疗法的一个重要副作用是细胞因子释放综合症--随着CAR-T细胞的繁殖,会引起细胞因子大量释放到血液中,严重情况下会引起患者死亡。

这项试验是由Carl June领导的宾夕法尼亚大学Abramson Cancer Center中心的研究人员完成。研究人员在基因编辑方法上进行了革新,他们没有在T细胞上添加新的受体,而是删除了三个基因,其中两个与T细胞受体有关,第三个为PD-1的编辑基因。

June在评论结果时说,来自患者的数据"证明了两个重要的事情,据我们所知,这是以前没有人证明过的。首先,我们可以在制备过程中成功地进行精确的多次编辑,所产生的细胞在人体中的存活时间比以前公布的任何数据所显示的时间都要长。第二,到目前为止,这些细胞显示出持续的攻击和杀死肿瘤的能力。"

去年11月,Vertex和CRISPR Therapeutics揭示了前两名接受研究性CRISPR / Cas9基因编辑疗法CTX001治疗患者的初步阳性数据。

与宾夕法尼亚州的研究不同,Vertex说其CRISPR治疗既安全又有效,两名患者已经被治愈。两名患者都有严重的血红蛋白病,包括输血依赖性β地中海贫血(TDT)和严重的镰状细胞病(SCD)。

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    2021-01-23 一叶知秋
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    2020-02-13 yuandd
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    2020-02-13 jichang