NEJM:慢病毒基因治疗联合低剂量白消安治疗SCID-X1婴儿

2019-04-18 xing.T 网络

由此可见,慢病毒载体基因治疗结合低暴露、靶向白消安调节对新诊断的SCID-X1婴儿具有低度急性毒性作用,在中位随访16个月期间,可出现转导细胞的多系列植入,功能性T细胞和B细胞的重建以及NK细胞计数正常化。

X-连锁严重联合免疫缺陷(SCID-X1)的异基因造血干细胞移植经常无法在匹配的兄弟捐赠者获得与T细胞、B细胞和自然杀伤(NK)细胞相关的免疫力,除非接受高剂量化疗。在先前的研究中,用γ-逆转录病毒载体进行的自体基因治疗未能重建B细胞和NK细胞免疫,并且由于载体相关的白血病而使病情复杂化。

近日,顶级医学期刊NEJM上发表了一篇研究文章,研究人员在8名新诊断的SCID-X1婴儿中进行了双中心1-2期试验以评估低暴露、靶向白消安调节后,通过慢病毒载体将IL2RG互补DNA转移至骨髓干细胞的安全性和功效。

研究人员对8例SCID-X1患儿进行了随访,中位数为16.4个月。骨髓收获、白消安调节和细胞输注没有意想不到的副作用。在7名婴儿中,输注后3至4个月CD3、CD4和幼稚CD4 T细胞和NK细胞的数量正常化,并伴有T细胞、B细胞、NK细胞、骨髓细胞和骨髓祖细胞中的载体标记 。第八名婴儿最初的T细胞计数不足,但在没有白消安调节的基因校正细胞增强后,T细胞在这个婴儿中发育。所有婴儿以前的感染均清除,并且所有婴儿都继续正常生长。 8名婴儿中有7名IgM水平正常化,其中4名患者停用静脉注射免疫球蛋白;这四个婴儿中有三个对疫苗有反应。对7名婴儿进行了载体插入位点分析,发生在所有7名患者中没有克隆优势的多克隆模式。

由此可见,慢病毒载体基因治疗结合低暴露、靶向白消安调节对新诊断的SCID-X1婴儿具有低度急性毒性作用,在中位随访16个月期间,可出现转导细胞的多系列植入,功能性T细胞和B细胞的重建以及NK细胞计数正常化。 

原始出处:

Ewelina Mamcarz,et al.Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.NEJM.2019.https://www.nejm.org/doi/full/10.1056/NEJMoa1815408

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    2020-01-30 wfang545
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    2019-12-23 xlxchina
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    2020-03-09 xugumin
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    2019-04-20 ymljack
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    2019-04-18 旺医

    顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息

    0

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