近日，CRISPR基因编辑技术先驱之一，Jennifer Doudna博士发表论文，剖析了另一团队先后发表在《Nature Methods》两篇前后结论相悖的论文。
此前认为CRISPR基因编辑存在严重缺陷，引起无法预测的突变。最新研究结论：CRISPR-Cas9编辑对基因组进行精确编辑，并不会引入大量非预期的“基因脱靶突变”。
基因编辑技术CRISPR的发明者之一Jennifer Doudna博士，去年7月专门撰文严厉抨击了发表在《Nature Methods》上一篇研究论文的观点。
该论文作者此前曾大肆宣扬CRISPR基因编辑技术的精确性，但在这篇文章中又反过来声称发现CRISPR基因编辑的严重缺陷，认为该技术可引起数百个无法预测的突变，容易被科学界所忽略。
该论文发表后，三家以基因编辑为核心技术的生物服务公司，Editas医药、Intellia Therapeutics和CRISPR Therapeutics的股票应声大跌。
与此同时，这几家公司的科学家们也立即申辩该文章相关研究方法和结论存在缺陷。Doudna博士查阅了发表在《Nature Methods》的论文和《BioRxiv》预印本中的数据分析结果之后，也认同科学家们的观点： CRISPR基因编辑技术并没有该作者在文章中所描述的那么夸张。
Doudna博士认为：科学会自我修正。该论文作者(Kellie Schaefer和同事们）重新梳理了CRISPR编辑中发现的所有脱靶突变，以预印本形式公布在《bioRxiv》在线版。
然而，得出基因编辑技术造成“严重脱靶”的结论，也与该团队最初的结果相悖，即CRISPR-Cas9编辑技术可以对基因组进行精确编辑，并不会诱发大量非预期的”脱靶突变”。
Doudna博士去年7月也在相关文章中表示，《 Nature Methods》急于发表一个有争议的结果、过于草率，或许是期刊编辑邀请同行评审不到位：或是没有找对审稿专家。
庆幸的是，该篇文章的作者也感受到了来自科学界的“争议”，他们重新审视了此前的研究结果。
近日《Nature Method》刊发的这篇新论文被认为是原论文的“勘误表”（更正）。终于，3月30日，原始论文在《 Nature Methods》官网上被撤回，并发表了联合作者们的各自观点。
这一切正像CRISPR技术的精髓：“修正错误”。
科学研究中出现技术或结论性错误，并非不可原谅，但是，造假实验数据或臆想结论，国内某些专家基因编辑“突破”成就，就不是用“勘误”所能更正的了。
原文参考：
1、Unexpected mutationsafter CRISPR-Cas9 editing in vivo.
SchaeferKA, WuWH, ColganDF, TsangSH, BassukAG, MahajanVB.
Nat Methods. 2017May 30;14(6):547-548.
doi: 10.1038/nmeth.4293.
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2、Corrigendum and follow-up: Whole genome sequencing ofmultiple CRISPR-edited mouse lines suggests no excess mutations.
Kellie A. Schaefer, Benjamin W. Darbro, DianaF. Colgan, Stephen H. Tsang, Alexander G. Bassuk, VinitB. Mahajan, doi: https://doi.org/10.1101/154450
3、CRISPR–Cas9 Structures and Mechanisms
Fuguo Jiang and Jennifer A. Doudna
AnnualReview of Biophysics, Vol. 46:505-529
https://doi.org/10.1146/annurev-biophys-062215-010822
https://doi.org/10.1146/annurev-biophys-062215-010822
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撤稿告示《Nature Method》Retracted online 30 March 2018
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This paper is being retracted because the genomic variants observed by the authors in two CRISPR-treated mice cannot be conclusively attributed to CRISPR–Cas9.The paper was a peer-reviewed Correspondence in the journal.The authors made their observation as part of their work on correction of a gene involved in blindness.The authors used mice of the inbred FVB/NJ strain from the JAX genetic quality control program that were purchased within months of each other and that were not bred in the authors’ laboratory.The assumption was that this design was sufficient to control for genetic variation in an inbred strain.Since publication of the work, however, it has been brought to the journal’s and the authors’ attention that without parental controls or more analysis of genetic background, it is not certain that the variants reported are due to CRISPR treatment
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(https://doi.org/10.1038/nmeth.4559, https://doi.org/10.1038/nmeth.4552, https://doi.org/10.1038/nmeth.4553, https://doi.org/10.1038/nmeth.4541, https://doi.org/10.1038/nmeth.4554).
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The study is therefore being retracted to maintain the accuracy of the scientific record.1 O4 b’ `; y- V t+ w/ Z r0 ~3 c
作者的观点
S.H.T. and W.-H.W. agree with the retraction.
S.H.T. and W.-H.W. agree with the retraction.
K.A.S., D.F.C., A.G.B. and V.B.M. do not agree with the retraction.
编辑部的意见：
All authors note that there is very little whole-genome sequencing data on the effects of CRISPR treatment in vivo. The question of whether CRISPR has effects on the in vivo genome will require further study; the authors are carrying out follow-up studies using whole-genome sequencing.