和病毒打基因战,利用CRISPR/Cas9剔除艾滋病基因

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一直以来让人们闻之色变的爱滋病,即将有新的治疗方式了。由Kamel Khalili带领的研究团队,在<<Gene Therapy>>上发表了一篇研究,这是他们第一次成功地将HIV病毒基因,从带有爱滋病病毒基因的老鼠基因组内去除。

HIV病毒DNA会永远嵌入病患感染细胞中

艾滋病全名为后天免疫缺乏症候群(Acquired Immune Deficiency Syndrome,AIDS),因人类免疫缺陷病毒(Human Immunodeficiency Virus,HIV)的感染所引起,HIV病毒是反转录病毒,在感染人类时会将自己的遗传物质反转录成为DNA,再嵌入人类细胞的DNA中,利用人类本身的系统,大量制作出病毒,破坏免疫系统,导致人体无法抵挡其他疾病。而嵌入人类DNA中的病毒基因则会一直潜伏在人类细胞中。

利用CRISPR/CAS9编修基因,剔除体内HIV病毒基因

由Kamel Khalili带领的研究团队尝试使用基因编辑的方式来治疗艾滋病。几年前,他们使用基因编辑工具 CRISPR/Cas9成功剔除来自艾滋病患的细胞。然而,只有细胞实验的数据尚不足以发展一种新的疗法,因此这次他们尝试使用活体动物来进行试验。他们使用大鼠(rats)及小鼠(mouse)两种模式动物进行试验,首先,研究团队设计了一段HIV的基因,透过分子技术,让老鼠体内几乎所有细胞都被嵌入HIV基因,模拟人类感染的情况,接着再设计针对HIV病毒基因的CRISPR/Cas9,将其透过老鼠尾部的静脉注射进入血液,利用基因编辑的方式,来改变HIV病毒感染的足迹。CRISPR厉害的地方在于,它能够十分精确地剔除目标病毒基因,并让动物体本身的DNA完好无缺。

治疗方式简单有效,期待临床试验能造福更多病患

他们的研究显示,CRISPR在大鼠及小鼠中剔除病毒基因的能力十分不错,在两种物种中,都能剔除一半以上的病毒基因。虽然无法完全去除HIV病毒的基因,但却能够大量减少病毒的感染。目前的抗艾滋药物虽有不错的效果,但同样无法去除所有的病毒。这个尚在实验阶段的研究,也许能够成为传统治疗方式很好的辅助方法。Kamel Khalili表示,这是很大的一步进展,他们正在进行更进一步的研究,好确定需要给予的CRISPR量,并且监控是否有出现任何的副作用。Kamel Khalili说道:“这真的十分简单,如果这项技术之后顺利进入临床治疗阶段,将能将治疗变得十分简单,不用透过骨髓移植等浩大又困难的手术,就能治愈艾滋病患者。”另外,若能顺利进入临床治疗,将能造福许多发展中国家的病患,因为大多数的艾滋病患身处于医疗资源较贫瘠的地区。

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