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利用CRISPR/CAS9技术在生殖细胞中治愈小鼠遗传疾病(英文)
引用本文:李大力.利用CRISPR/CAS9技术在生殖细胞中治愈小鼠遗传疾病(英文)[J].中国科学通报(英文版),2015(3):400-402.
作者姓名:李大力
作者单位:Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University
摘    要:The CRISPR/Cas system is a very powerful and versatile tool for genomic manipulation.A recent study published in Cell Research by Wu et al.1]reported a Cas9-mediated strategy to correct a disease-causing mutation in the mouse gamma C-crystallin(Crygc)locus in spermatogonial stem cells(SSCs)to cure the resulting cataracts disease in the offspring with 100%efficiency.Importantly,no off-target mutations were identified through whole-genome sequencing.

关 键 词:spermatogonial  遗传疾病  CRISPR/CAS9  sequencing  offspring  genomic  powerful  manipulation  gamma  locus
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