2015年11月5日英国包括<<每日邮报>>、 <<镜报>>在内的多家报纸报道了世界上首例婴儿白血病患者被成功治愈的奇迹。2014年年6月，可爱的Layla Richards 诞生于伦敦的北部，然而三个月后，Layla开始患病，医生通过血液检测将其确诊为急性淋巴白血病患者。疾病确诊后，Layla 接受了化疗，骨髓移植等传统治疗方法，但不幸的是这些治疗方法对于Layla的病情缓解并没有帮助。在医生宣布了Layla的治疗无望后，Layla的父母却并没有放弃。相反，他们允许伦敦Great Ormond Street医院的医生采用德国生物技术公司Cellectis开创的细胞治疗方法治疗Layla。事实上这种方法尚没有应用在临床，之前仅在老鼠身上做过测试。今年6月份，医生将1毫升基因编辑过的5000万个细胞注入了Layla的体内。奇迹发生了，几周后Layla 开始出现好转的迹象，如今检测结果发现Layla身体内的癌细胞已经完全消失了。
基因编辑技术如何创造奇迹？
那么医生具体是怎么对Layla进行治疗的呢？医生给Layal注射的血细胞来自美国的一个随机的捐献者。科学家对这些细胞进行了三种形式的修改。首先，这些血细胞内被注入了抗白血病基因，这些基因可以编码靶向追踪并杀死癌细胞的蛋白质。其次，科学家关闭了血细胞内的两个基因，关闭其中一个基因是为了确保注射的细胞不被Layla 身体排斥，关闭的另一个基因是为了确保捐赠的细胞不被药物杀死。今年6月份，医生将编辑过的血细胞注入Layla体内，经过三个月，这些细胞寻找并杀死了癌症细胞。9月份，医生又重新对Layla进行了骨髓移植，新的骨髓重新开始生产健康的血液细胞，而捐献的细胞在骨髓移植过程中将会丧生。
基因编辑技术的新宠
在Layla治疗过程，基因工程编辑过的细胞无疑是最重要的因素。那么基因是如何被编辑的呢？ 注射到Layla体内的细胞是通过一种叫做TALENs的新型基因编辑技术进行的。这项技术极为复杂，普通的实验室是无法进行的，只有借助商业公司完成。小编想在这里介绍有第三代基因编辑技术之称得GRISPR/Cas9系统。
所谓基因编辑技术，是指对DNA核苷酸序列进行删除和插入等操作。CRISPR-Cas9系统并非天生就是为人类使用而产生的。它的本质其实是细菌中一种对付诸如病毒等外来DNA的防御系统。CRISPR（Clustered Regularly Interspaced Short Palindromic Repeats，CRISPR）是生命进化历史上，细菌和病毒进行斗争产生的免疫武器，简单说就是病毒能把自己的基因整合到细菌，利用细菌的细胞工具为自己的基因复制服务，细菌为了将病毒的外来入侵基因清除，进化出CRISPR系统，利用这个系统，细菌可以不动声色地把病毒基因从自己的染色体上切除，这是细菌特有的免疫系统。微生物学家10年前就掌握了细菌拥有多种切除外来病毒基因的免疫功能，其中比较典型的模式是依靠一个复合物，该复合物能在一段RNA指导下，定向寻找目标DNA序列，然后将该序列进行切除。许多细菌免疫复合物都相对复杂，其中科学家掌握了对一种蛋白Cas9的操作技术，并先后对多种目标细胞DNA进行切除。这种技术被称为CRISPR/Cas9基因编辑系统，迅速成为生命科学最热门的技术。
CRISPR/Cas9应用前景
在过去的3年里，基因编辑神器CRISPR被认为是遗传研究领域的革命性技术；科学家用它来编辑农作物、家畜甚至是人类胚胎。一些公司如雨后春笋般出现，开发基于CRISPR的基因疗法。有科学家表示，此类疗法的首次临床试验会在接下来的一到两年内进行。这些首批试验或许将勾勒出CRISPR的应用场景，即CRISPR成分能被直接注入眼睛等器官，或者细胞能从人体移除并在实验室中进行基因改造后被放回体内。例如，形成血液的干细胞可能被修正用于治疗诸如镰状细胞性贫血症或β-地中海贫血等疾病。虽然将酶和引导性RNA送入很多其他组织将是一项更大的挑战，但研究人员希望有一天这项技术能被用于解决更广范的遗传疾病。
CRISPR/Cas9技术是一项正在发展中的新兴技术，仍然存在一些缺陷，如脱靶效应，系统效率和特异性受不同物种或同一物种的不同基因影响等因素。然而，作为一项新技术，纵然还有许多问题有待解决与发展，也给研究人员以更广阔空间去进行探索和研究，不断发现、发展该项技术的更多更大的优势。未来，CRISPR/Cas技术会在实验室中得到广泛的推广，并成为科研人员了解生物、在改造基因，解决更多的问题的一项常规的应用手段。
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