In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

Swiech, Lukasz, Heidenreich, Matthias, Banerjee, Abhishek, Habib, Naomi, Li, Yinqing, Trombetta, John, Sur, Mriganka, Zhang, Feng·2015·Nature Biotechnology

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  10. CRISPR–Cas9-mediated genome editing and guide RNA design
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  53. Education and the Environment: Learning To Live with Limits.
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  54. CRISPR-Cas9-mediated genome editing in one blastomere of two-cell embryos reveals a novel Tet3 function in regulating neocortical development
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