本文摘要：Scientists have successfully tweaked the DNA of mice with a specific genetic mutation to prevent them from going completely deaf. If the gene-editing technique is proven safe, it could one day be used to treat the same type of hearing loss

Scientists have successfully tweaked the DNA of mice with a specific genetic mutation to prevent them from going completely deaf. If the gene-editing technique is proven safe, it could one day be used to treat the same type of hearing loss in people.科学家已顺利调整了装载特定基因突变的小鼠的DNA，以防治它们几乎失明。如果能证明基因编辑技术是安全性的，那么有朝一日这一方法或能用作化疗人类完全相同类型的听力损失。

Researchers injected the gene-editing tool CRISPR-Cas9 inside the ears of live mice with a deafness-causing genetic mutation. The molecular scissors were able to precisely cut the disease-causing copy of the gene without disrupting the healthy copy, according to a study published today in Nature. Even though the researchers think they were able to repair only a small fraction of cells in the ear that prevented treated mice from losing all their hearing.研究人员将基因编辑工具CRISPR-Cas9静脉注射到活的小鼠的耳内，这些小鼠装载耳聋基因突变。《大自然》期刊于今日公开发表的一项研究指出：分子剪刀需要准确的切割成可怕基因的拷贝，而且会毁坏身体健康拷贝。

尽管研究人员指出他们不能修缮耳内的一小部分细胞，以避免被化疗的小鼠几乎失明。Gene editing has been making huge strides in the past few years. Just last month, scientists attempted to edit a persons DNA inside his own body for the first time in order to cure a debilitating genetic disorder called Hunter syndrome. The technique described in todays study also attempts to edit DNA inside the body of a living animal - in this case, mice. Though the treatment is still years from coming to a clinic near you, its an important step in the development of gene therapies, which tinker with genes in order to treat or prevent diseases.基因编辑在过去几年里获得了长足进展。

就在上个月，科学家首次尝试在人体内编辑自己的DNA，以化疗一种被称作亨特氏综合症的疲惫遗传疾病。今天研究中叙述的这项技术也企图编辑活体动物体内的DNA--在这种情况下，也就是小鼠。

尽管这一化疗方法有可能还必须好几年的时间才能应用于临床，但这是基因疗法发展中的最重要一步，这是一种标记基因以化疗或防治疾病的疗法。We have entered the age where the human genome is a real drug target, says Fyodor Urnov, the associate director at Altius Institute for Biomedical Sciences, who was not involved in the study. The researchers have provided the first important step and a strong perspective of hope for people who have this mutation.我们早已转入了人类基因组确实沦为药物标靶的时代，Altius Institute for Biomedical Sciences的副所长费奥多·乌尔诺夫说，他没参予到这项研究。研究人员为装载这种变异的人获取了最重要的第一步、为他们带给了强劲的期望。

The gene-editing tool CRISPR-Cas9 is based on a defense mechanism bacteria use to ward off viruses by cutting off bits of their DNA. Scientists have engineered that mechanism to edit pieces of the genetic code, creating unusually muscular beagles, for instance, and mosquitoes that dont transmit malaria. The technique is advancing fast: Last year in China, doctors took immune cells from a patient with lung cancer, edited them, and then injected the cells back into the patient to help defeat the disease.基因编辑工具CRISPR-Cas9创建在防御机制细菌的用于基础之上，通过截断DNA的位点以抵挡病毒。科学家设计了这种机制来编辑遗传密码，例如建构出现异常的肌肉小猎犬、建构不传播疟疾的蚊子。这一技术正在飞速发展：去年在中国，医生从肺癌患者体内拿走了免疫细胞、对这些细胞展开编辑，然后将其新的注回患者体内，以协助他们战胜肺癌。

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