2024年3月19日，荷兰阿姆斯特丹大学的研究人员在一次医学会议上介绍称，他们通过使用CRISPR基因技术（获得2020年诺贝尔化学奖）已成功从被感染的细胞中清除了HIV病毒（人类免疫缺陷病毒，即艾滋病毒）。

该研究原计划于今年4月27日至30日在西班牙巴塞罗那举行的欧洲临床微生物学和传染病大会（ECCMID 2024）发表。

相关研究由荷兰阿姆斯特丹大学医学中心的埃琳娜·埃雷拉-卡里略（Elena Herrera-Carrillo）博士及其团队成员包元玲（音）、于正浩（音）和帕斯卡·克鲁恩（Pascal Kroon）领导。

关于CRISP技术

CRISPR技术是一种基因技术，类似于一把能切割DNA的剪刀，在向导RNA（gRNA）的引导下，可以剪切掉部分“坏掉”的基因片段或使其失活，从而实现对特定基因序列的精确和修饰。这一行为既有利于删除不需要的基因，也有利于将新的遗传物质引入生物体细胞，从而为先进的治疗方法铺平道路。其发明者珍妮弗·杜德纳和艾曼纽尔·查彭蒂耶凭借这项突破性技术获得了2020年的诺贝尔化学奖。

HIV治疗的重大挑战之一是该病毒具有将自身基因组整合到宿主DNA中的能力，尽管目前有多种有效的抗病毒药物用于治疗HIV感染，但只能抑制HIV在人体内的复制，无法将其清除，故患者需要接受终身抗病毒治疗，因为一旦抗病毒治疗停止，HIV可能会卷土重来。

HIV可以感染体内不同类型的细胞和组织，每种细胞和组织都有其独特的环境和特征。荷兰研究人员对此表示， CRISPR-Cas的功能就像“分子剪刀”一样，在向导RNA (gRNA) 的指导下，可以在指定点切割DNA，他们正在寻找一种在所有这些情况下都可灭活艾滋病毒的方法，“我们的目标是开发一种强大且安全的组合CRISPR-Cas方案，可以在不同的细胞环境中灭活不同的艾滋病毒毒株。”    

既往曾有科学家利用 CRISPR 技术成功

地从人类细胞中彻底清除了潜在HIV-1病毒

早在十余年前，来自美国 Temple University 的研究团队就曾利用 CRISPR/Cas9 基因技术，将人体免疫反应中扮演重要角色的T细胞上的 HIV-1 病毒移除，使其无法再进一步的感染其他健康细胞。该项研究成果发表在美国《国家科学院学报》上。

CRISPR技术治疗艾滋病仍处于“概念验证”阶段

阿姆斯特丹大学的科学家们还强调，目前，他们的科研进程仍然处于“概念验证”阶段，并不会很快成为成熟的HIV治愈疗法，并且还需要通过大量工作来确保这种方法安全且有效。但是他们相信，使用CRISPR技术后，未来有望实现完全清除患者体内病毒的目标。

伦敦弗朗西斯·克里克研究所的病毒专家乔纳森·斯托伊博士表示，从可能携带HIV的所有细胞中清除HIV是“极具挑战性”的一项工作。因为“治疗后的非特异性作用（治疗行为在靶标以外的地方产生的影响或效果）和可能的长期副作用仍然是一个令人担忧的痛点”。因此即使假设这种治疗能够被证明有效，也很可能需要很多年的时间，任何基于CRISPR的治疗才会变得常规化。

诺丁汉大学的干细胞和基因治疗技术副教授詹姆斯·迪克森博士也同意这一观点，他表示，“今后仍然需要做更多的工作来证明这些细胞含量测定的结果可以在整个身体中都能实现，以便推进未来的治疗方案进步。”

撰文 | Anna.An

| Swagpp

版权说明：梅斯医学（MedSci）是国内领先的医学科研与学术服务平台，致力于医疗质量的改进，为临床实践提供智慧、精准的决策支持，让医生与患者受益。欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。

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