In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9 (original) (raw)
Hsu, P.D., Lander, E.S. & Zhang, F. Development and applications of CRISPR-Cas9 for genome engineering. Cell157, 1262–1278 (2014). ArticleCAS Google Scholar
Xue, W. et al. CRISPR-mediated direct mutation of cancer genes in the mouse liver. Nature 10.1038/nature13589 (6 August 2014).
Yin, H. et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat. Biotechnol.32, 551–553 (2014). ArticleCAS Google Scholar
Ding, Q. et al. Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing. Circ. Res.115, 488–492 (2014). ArticleCAS Google Scholar
Wu, Z., Yang, H. & Colosi, P. Effect of genome size on AAV vector packaging. Mol. Ther.18, 80–86 (2010). ArticleCAS Google Scholar
Gray, S.J. et al. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum. Gene Ther.22, 1143–1153 (2011). ArticleCAS Google Scholar
Ostlund, C. et al. Dynamics and molecular interactions of linker of nucleoskeleton and cytoskeleton (LINC) complex proteins. J. Cell Sci.122, 4099–4108 (2009). ArticleCAS Google Scholar
Chahrour, M. & Zoghbi, H.Y. The story of Rett syndrome: from clinic to neurobiology. Neuron56, 422–437 (2007). ArticleCAS Google Scholar
Chen, R.Z., Akbarian, S., Tudor, M. & Jaenisch, R. Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in mice. Nat. Genet.27, 327–331 (2001). ArticleCAS Google Scholar
Li, Y. et al. Global transcriptional and translational repression in human-embryonic-stem-cell-derived Rett syndrome neurons. Cell Stem Cell13, 446–458 (2013). Article Google Scholar
Zhou, Z. et al. Brain-specific phosphorylation of MeCP2 regulates activity-dependent Bdnf transcription, dendritic growth, and spine maturation. Neuron52, 255–269 (2006). ArticleCAS Google Scholar
Qi, L.S. et al. Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression. Cell152, 1173–1183 (2013). ArticleCAS Google Scholar
Bikard, D. et al. Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system. Nucleic Acids Res.41, 7429–7437 (2013). ArticleCAS Google Scholar
Gilbert, L.A. et al. CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell154, 442–451 (2013). ArticleCAS Google Scholar
Grindberg, R.V. et al. RNA-sequencing from single nuclei. Proc. Natl. Acad. Sci. USA110, 19802–19807 (2013). ArticleCAS Google Scholar
Moretti, P. et al. Learning and memory and synaptic plasticity are impaired in a mouse model of Rett syndrome. J. Neurosci.26, 319–327 (2006). ArticleCAS Google Scholar
Kheirbek, M.A. et al. Differential control of learning and anxiety along the dorsoventral axis of the dentate gyrus. Neuron77, 955–968 (2013). ArticleCAS Google Scholar
Chahrour, M. et al. MeCP2, a key contributor to neurological disease, activates and represses transcription. Science320, 1224–1229 (2008). ArticleCAS Google Scholar
Hubel, D.H. & Wiesel, T.N. Receptive fields of single neurones in the cat's striate cortex. J. Physiol. (Lond.)148, 574–591 (1959). ArticleCAS Google Scholar
Banerjee, A., Castro, J. & Sur, M. Rett syndrome: genes, synapses, circuits, and therapeutics. Front. Psychiatry3, 34 (2012). ArticleCAS Google Scholar
Chao, H.T., Zoghbi, H.Y. & Rosenmund, C. MeCP2 controls excitatory synaptic strength by regulating glutamatergic synapse number. Neuron56, 58–65 (2007). ArticleCAS Google Scholar
Wood, L., Gray, N.W., Zhou, Z., Greenberg, M.E. & Shepherd, G.M. Synaptic circuit abnormalities of motor-frontal layer 2/3 pyramidal neurons in an RNA interference model of methyl-CpG-binding protein 2 deficiency. J. Neurosci.29, 12440–12448 (2009). ArticleCAS Google Scholar
McGraw, C.M., Samaco, R.C. & Zoghbi, H.Y. Adult neural function requires MeCP2. Science333, 186 (2011). ArticleCAS Google Scholar
Feng, J. et al. Dnmt1 and Dnmt3a maintain DNA methylation and regulate synaptic function in adult forebrain neurons. Nat. Neurosci.13, 423–430 (2010). ArticleCAS Google Scholar
Fu, Y. et al. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat. Biotechnol.31, 822–826 (2013). ArticleCAS Google Scholar
Hsu, P.D. et al. DNA targeting specificity of RNA-guided Cas9 nucleases. Nat. Biotechnol.31, 827–832 (2013). ArticleCAS Google Scholar
Cong, L. et al. Multiplex genome engineering using CRISPR/Cas systems. Science339, 819–823 (2013). ArticleCAS Google Scholar
Fonfara, I. et al. Phylogeny of Cas9 determines functional exchangeability of dual-RNA and Cas9 among orthologous type II CRISPR-Cas systems. Nucleic Acids Res.42, 2577–2590 (2014). ArticleCAS Google Scholar
Esvelt, K.M. et al. Orthogonal Cas9 proteins for RNA-guided gene regulation and editing. Nat. Methods10, 1116–1121 (2013). ArticleCAS Google Scholar
Platt, R.J. et al. CRISPR-Cas9 knockin mice for genome editing and cancer modeling. Cell159, 440–455 (2014). ArticleCAS Google Scholar
Levitt, N., Briggs, D., Gil, A. & Proudfoot, N.J. Definition of an efficient synthetic poly(A) site. Genes Dev.3, 1019–1025 (1989). ArticleCAS Google Scholar
Jinek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science337, 816–821 (2012). ArticleCAS Google Scholar
Sapranauskas, R. et al. The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli. Nucleic Acids Res.39, 9275–9282 (2011). ArticleCAS Google Scholar
McClure, C., Cole, K.L., Wulff, P., Klugmann, M. & Murray, A.J. Production and titering of recombinant adeno-associated viral vectors. J. Vis. Exp.57, e3348 (2011). Google Scholar
Konermann, S. et al. Optical control of mammalian endogenous transcription and epigenetic states. Nature500, 472–476 (2013). ArticleCAS Google Scholar
Banker, G. & Goslin, K. Developments in neuronal cell culture. Nature336, 185–186 (1988). ArticleCAS Google Scholar
Swiech, L. et al. CLIP-170 and IQGAP1 cooperatively regulate dendrite morphology. J. Neurosci.31, 4555–4568 (2011). ArticleCAS Google Scholar
Sholl, D.A. Dendritic organization in the neurons of the visual and motor cortices of the cat. J. Anat.87, 387–406 (1953). CASPubMedPubMed Central Google Scholar
Hagihara, H., Toyama, K., Yamasaki, N. & Miyakawa, T. Dissection of hippocampal dentate gyrus from adult mouse. J. Vis. Exp.33, 1543 (2009). Google Scholar
Ran, F.A. et al. Genome engineering using the CRISPR-Cas9 system. Nat. Protoc.8, 2281–2308 (2013). ArticleCAS Google Scholar
Picelli, S. et al. Smart-seq2 for sensitive full-length transcriptome profiling in single cells. Nat. Methods10, 1096–1098 (2013). ArticleCAS Google Scholar
Fujita, P.A. et al. The UCSC Genome Browser database: update 2011. Nucleic Acids Res.39, D876–D882 (2011). ArticleCAS Google Scholar