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Brave New Genome 勇创新基因组 | 标准美音

VOM 国际医声
2015-07-25原文

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Brave New Genome

A Perspective article by Eric Lander, from the Broad Institute


of MIT and Harvard, Cambridge, Massachusetts.

Genome editing ( 基 因 组 编 辑 ) holds great therapeutic


promise. To treat HIV infection, physicians might edit
apatient's immune cells to delete the CCR5 gene, conferring
the resistance to HIV carried by the 1% of the U.S. population
lacking functional copies of this gene. To treat progressive
blindness (渐进性失明)caused by dominant (显性) forms
of retinitis pigmentosa ( 视 网 膜 色 素 变 性 ) ,they might
inactivate the mutant (突变型) allele (等位基因)in retinal
cells.

These goals will require overcoming serious technical


challenges such as avoiding “off-target” (偏离目标的) edits
elsewhere in the genome, which might give rise to cancer.
However, the technology also raises a more troubling
possibility: creating children carrying permanent, heritable
changes to the human germline DNA. The press has dubbed
such brave new progeny“designer babies” ( 品 牌 婴 孩 ) or
“genetically modified humans.”(转基因人)

The task now is to develop a clear framework for evaluating


human germline editing.

In this article, the author offers a starting point, focusing on


four key issues.

The first is technical: whether genome editing can be


performed with sufficient precision ( 精 确 度 ) to permit
scientists to responsibly contemplate creating genetically
modified babies.

The second issue is whether there are compelling


(非常强烈的) medical needs that outweigh the risks — both
from inaccurate editing and from unanticipated effects of the
intended edits.

The third key issue is who has the right todecide.

The final issue concerns morality (伦理道德)— what's right


and wrong and how we ought to live as a society.

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)于2015年7月23日,发布了第117号《国家食品药品监督管理总局关于开展药物临床
试验数据自查核查工作的公告》,药物研发企业面临短期内(8月下旬结束)开展临
床试验数据自查工作的巨大压力。北京美康福瑞医药科技有限公司现联合具有丰富数
据自查核查工作的美籍资深华人生物医学数据统计师团队,可接受委托承担药物临床
试验的统计分析和试验数据的自查和核查工作。业务联络,请联系在北京的谭亚娣博
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Copyright statement: The audio originates from NEJM.org; the text is


prepared by "Voice of Medicine(VOM)" work group.
音 频 和 英 文 文 字 源 自 NEJM.org 网 站 ,
由“国际医声”工作组编辑并添加中文注释。未经允许不得转载。
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