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最新一期（2013.12.5）cell stem cell发表了中科院李劲松课题组的最新科研成果：用crispr技术纠正基因突变引起的白内障获得了成功。2012年以来，Crispr以迅雷不及掩耳响叮当之势成为了强大的基因编辑工具，由于其简单易用和高效成为基因敲除/敲入的常用手段。在这篇文章中，作者将cas9 mRNA和sgRNA注射到Crygc突变的小鼠受精卵，通过同源序列指导的DNA修复来纠正发生突变的碱基序列，使得突变的Crygc基因序列恢复正常，从而使新生小鼠免于此基因导致的白内障。
8 O4 n5 J5 t, U2 P% S% E% D以下为文章的摘要：! y) |- c0 l+ C; N
The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease. |
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发表于 2013-12-9 20:56
发表于 2013-12-9 21:27
进展好快
