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.
科学家已成功调整了携带特定基因突变的小鼠的DNA，以预防它们完全失聪。如果能证明基因编辑技术是安全的，那么有朝一日这一方法或能用于治疗人类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注射到活的小鼠的耳内，这些小鼠携带耳聋基因突变。《自然》期刊于今日发表的一项研究表明：分子剪刀能够精确的切割致命基因的拷贝，而且不会破坏健康拷贝。尽管研究人员认为他们只能修复耳内的一小部分细胞，以防止被治疗的小鼠完全失聪。
Gene editing has been making huge strides in the past few years. Just last month, scientists attempted to edit a person's DNA inside his own body for the first time in order to cure a debilitating genetic disorder called Hunter syndrome. The technique described in today's 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, it's 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取得了长足进展。就在上个月，科学家首次尝试在人体内编辑自己的DNA，以治疗一种被称作亨特氏综合症的虚弱遗传疾病。今天研究中描述的这项技术也试图编辑活体动物体内的DNA--在这种情况下，也就是小鼠。尽管这一治疗方法可能还需要好几年的时间才能应用于临床，但这是基因疗法发展中的重要一步，这是一种修饰基因以治疗或预防疾病的疗法。
"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."
"我们已经进入了人类基因组真正成为药物标靶的时代，"Altius Institute for Biomedical Sciences的副所长费奥多·乌尔诺夫说道，他没有参与到这项研究。研究人员"为携带这种突变的人提供了重要的第一步、为他们带来了强大的希望。"
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 don't 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建立在防御机制细菌的使用基础之上，通过切断DNA的位点以抵御病毒。科学家设计了这种机制来编辑遗传密码，例如创造异常的肌肉小猎犬、创造不传播疟疾的蚊子。这一技术正在飞速发展：去年在中国，医生从肺癌患者体内取走了免疫细胞、对这些细胞进行编辑，然后将其重新注回患者体内，以帮助他们战胜肺癌。
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