In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
Open accessAbstract
Probing gene function in the mammalian brain can be greatly assisted with methods to manipulate the genome of neurons in vivo. The clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated endonuclease (Cas)9 from Streptococcus pyogenes (SpCas9) can be used to edit single or multiple genes in replicating eukaryotic cells, resulting in frame-shifting insertion/deletion (indel) mutations and subsequent protein depletion. Here, we delivered SpCas9 and guide RNAs using adeno-associated viral (AAV) vectors to target single (Mecp2) as well as multiple genes (Dnmt1, Dnmt3a and Dnmt3b) in the adult mouse brain in vivo. We characterized the effects of genome modifications in postmitotic neurons using biochemical, genetic, electrophysiological and behavioral readouts. Our results demonstrate that AAV-mediated SpCas9 genome editing can enable reverse genetic studies of gene function in the brain.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Adenovirus or AAV-mediated CRISPR-Cas9 delivery has been successfully applied to knockout genes in the mouse brain and liver.
“Adenovirus or adeno-associated virus (AAV)-mediated CRISPR-Cas9 delivery has been successfully applied to knockout genes in the mouse brain and liver12,13.”
From Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo (2016) · cites this paper as [13]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116 · see citing works
Multiplex Genome Engineering Using CRISPR/Cas Systems
Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339 · see citing works
Self-citationGenome engineering using the CRISPR-Cas9 system
Ran, F. A., Hsu, P. D., Wright, J., Agarwala, V., Scott, D. A., & Zhang, F. (2013) · Nature Protocols · doi:10.1038/nprot.2013.143 · Cited by 11,133 · see citing works
Self-citationDevelopment and Applications of CRISPR-Cas9 for Genome Engineering
Hsu, P. D., Lander, E. S., & Zhang, F. (2014) · Cell · doi:10.1016/j.cell.2014.05.010 · Cited by 5,762 · see citing works
Self-citationRepurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression
Qi, L. S., Larson, M. H., Gilbert, L. A., Doudna, J. A., Weissman, J. S., Arkin, A. P., & Lim, W. A. (2013) · Cell · doi:10.1016/j.cell.2013.02.022 · Cited by 5,016 · see citing works
DNA targeting specificity of RNA-guided Cas9 nucleases
Hsu, P. D., Scott, D. A., Weinstein, J. A., Ran, F. A., Konermann, S., Agarwala, V., Li, Y., Fine, E. J., Wu, X., Shalem, O., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2647 · Cited by 4,741 · see citing works
Self-citationReceptive fields of single neurones in the cat's striate cortex
Hubel, D. H., & Wiesel, T. N. (1959) · The Journal of Physiology · doi:10.1113/jphysiol.1959.sp006308 · Cited by 4,653 · see citing works
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Gilbert, L. A., Larson, M. H., Morsut, L., Liu, Z., Brar, G. A., Torres, S. E., Stern-Ginossar, N., Brandman, O., Whitehead, E. H., Doudna, J. A., et al. (2013) · Cell · doi:10.1016/j.cell.2013.06.044 · Cited by 3,696 · see citing works
High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
Fu, Y., Foden, J. A., Khayter, C., Maeder, M. L., Reyon, D., Joung, J. K., & Sander, J. D. (2013) · Nature Biotechnology · doi:10.1038/nbt.2623 · Cited by 3,292 · see citing works
Smart-seq2 for sensitive full-length transcriptome profiling in single cells
Picelli, S., Björklund, Å. K., Faridani, O. R., Sagasser, S., Winberg, G., & Sandberg, R. (2013) · Nature Methods · doi:10.1038/nmeth.2639 · Cited by 2,358 · see citing works
Dendritic organization in the neurons of the visual and motor cortices of the cat.
Sholl, D. A. (1953) · Europe PMC (PubMed Central) · Cited by 2,204 · see citing works
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
Platt, R. J., Chen, S., Zhou, Y., Yim, M. J., Swiech, L., Kempton, H. R., Dahlman, J. E., Parnas, O., Eisenhaure, T. M., Jovanovic, M., et al. (2014) · Cell · doi:10.1016/j.cell.2014.09.014 · Cited by 2,001 · see citing works
Self-citationThe UCSC Genome Browser database: update 2011
Fujita, P. A., Rhead, B. L., Zweig, A. S., Hinrichs, A. S., Karolchik, D., Cline, M., Goldman, M. J., Barber, G. P., Clawson, H., Coelho, A., et al. (2010) · Nucleic Acids Research · doi:10.1093/nar/gkq963 · Cited by 1,836 · see citing works
MeCP2, a Key Contributor to Neurological Disease, Activates and Represses Transcription
Chahrour, M., Jung, S. Y., Shaw, C., Zhou, X., Wong, S. T. C., Qin, J., & Zoghbi, H. Y. (2008) · Science · doi:10.1126/science.1153252 · Cited by 1,823 · see citing works
Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in mice
Chen, R. Z., Akbarian, S., Tudor, M., & Jaenisch, R. (2001) · Nature Genetics · doi:10.1038/85906 · Cited by 1,275 · see citing works
The Story of Rett Syndrome: From Clinic to Neurobiology
Chahrour, M., & Zoghbi, H. Y. (2007) · Neuron · doi:10.1016/j.neuron.2007.10.001 · Cited by 1,265 · see citing works
Bikard, D., Jiang, W., Samai, P., Hochschild, A., Zhang, F., & Marraffini, L. A. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt520 · Cited by 1,111 · see citing works
Self-citationEffect of genome size on AAV vector packaging
Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy · doi:10.1038/mt.2009.255 · Cited by 1,025 · see citing works
Dnmt1 and Dnmt3a maintain DNA methylation and regulate synaptic function in adult forebrain neurons
Feng, J., Zhou, Y., Campbell, S. L., Le, T., Li, E., Sweatt, J. D., Silva, A. J., & Fan, G. (2010) · Nature Neuroscience · doi:10.1038/nn.2514 · Cited by 1,019 · see citing works
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014) · Nature Biotechnology · doi:10.1038/nbt.2884 · Cited by 951 · see citing works
Zhou, Z., Hong, E. J., Cohen, S., Zhao, W.-N., Ho, H.-Y. H., Schmidt, L., Chen, W. G., Lin, Y., Savner, E., Griffith, E. C., et al. (2006) · Neuron · doi:10.1016/j.neuron.2006.09.037 · Cited by 877 · see citing works
The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli
Sapranauskas, R., Gasiūnas, G., Fremaux, C., Barrangou, R., Horvath, P., & Šikšnys, V. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr606 · Cited by 835 · see citing works
Optical control of mammalian endogenous transcription and epigenetic states
Konermann, S., Brigham, M. D., Trevino, A. E., Hsu, P. D., Heidenreich, M., Cong, L., Platt, R. J., Scott, D. A., Church, G. M., & Zhang, F. (2013) · Nature · doi:10.1038/nature12466 · Cited by 803 · see citing works
Self-citationOrthogonal Cas9 proteins for RNA-guided gene regulation and editing
Esvelt, K. M., Mali, P., Braff, J. L., Moosburner, M., Yaung, S. J., & Church, G. M. (2013) · Nature Methods · doi:10.1038/nmeth.2681 · Cited by 786 · see citing works
CRISPR-mediated direct mutation of cancer genes in the mouse liver
Xue, W., Chen, S., Yin, H., Tammela, T., Papagiannakopoulos, T., Joshi, N. S., Cai, W., Yang, G., Bronson, R., Crowley, D. G., et al. (2014) · Nature · doi:10.1038/nature13589 · Cited by 776 · see citing works
Self-citationDifferential Control of Learning and Anxiety along the Dorsoventral Axis of the Dentate Gyrus
Kheirbek, M. A., Drew, L. J., Burghardt, N. S., Costantini, D. O., Tannenholz, L., Ahmari, S. E., Zeng, H., Fenton, A. A., & Hen, R. (2013) · Neuron · doi:10.1016/j.neuron.2012.12.038 · Cited by 713 · see citing works
Learning and Memory and Synaptic Plasticity Are Impaired in a Mouse Model of Rett Syndrome
Moretti, P., Levenson, J. M., Battaglia, F., Atkinson, R., Teague, R., Antalffy, B., Armstrong, D., Arancio, O., Sweatt, J. D., & Zoghbi, H. Y. (2006) · The Journal of Neuroscience · doi:10.1523/jneurosci.2623-05.2006 · Cited by 545 · see citing works
Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing
Ding, Q., Strong, A., Patel, K. M., Ng, S.-L., Gosis, B. S., Regan, S. N., Cowan, C. A., Rader, D. J., & Musunuru, K. (2014) · Circulation Research · doi:10.1161/circresaha.115.304351 · Cited by 522 · see citing works
MeCP2 Controls Excitatory Synaptic Strength by Regulating Glutamatergic Synapse Number
Chao, H.-T., Zoghbi, H. Y., & Rosenmund, C. (2007) · Neuron · doi:10.1016/j.neuron.2007.08.018 · Cited by 511 · see citing works
RNA-sequencing from single nuclei
Grindberg, R. V., Yee-Greenbaum, J. L., McConnell, M. J., Novotny, M., O’Shaughnessy, A. L., Lambert, G. M., Araúzo-Bravo, M. J., Lee, J., Fishman, M., Robbins, G. E., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1319700110 · Cited by 422 · see citing works
Gray, S. J., Foti, S. B., Schwartz, J. W., Bachaboina, L., Taylor-Blake, B., Coleman, J., Ehlers, M. D., Zylka, M. J., McCown, T. J., & Samulski, R. J. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.245 · Cited by 420 · see citing works
Fonfara, I., Rhun, A. L., Chylinski, K., Makarova, K. S., Lécrivain, A.-L., Bzdrenga, J., Koonin, E. V., & Charpentier, E. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt1074 · Cited by 329 · see citing works
Li, Y., Wang, H., Muffat, J., Cheng, A. W., Orlando, D. A., Lovén, J., Kwok, S., Feldman, D., Bateup, H. S., Gao, Q., et al. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.09.001 · Cited by 305 · see citing works
Self-citationDefinition of an efficient synthetic poly(A) site.Preprint
Levitt, N. C., Briggs, D., Gil, A., & Proudfoot, N. J. (1989) · Genes & Development · doi:10.1101/gad.3.7.1019 · Cited by 290 · see citing works
Adult Neural Function Requires MeCP2
McGraw, C. M., Samaco, R. C., & Zoghbi, H. Y. (2011) · Science · doi:10.1126/science.1206593 · Cited by 255 · see citing works
Dissection of Hippocampal Dentate Gyrus from Adult Mouse
Hagihara, H., Toyama, K., Yamasaki, N., & Miyakawa, T. (2009) · Journal of Visualized Experiments · doi:10.3791/1543 · Cited by 203 · see citing works
Developments in neuronal cell culture
Banker, G., & Goslin, K. (1988) · Nature · doi:10.1038/336185a0 · Cited by 195 · see citing works
Östlund, C., Folker, E. S., Choi, J. C., Gomes, E. R., Gundersen, G. G., & Worman, H. J. (2009) · Journal of Cell Science · doi:10.1242/jcs.057075 · Cited by 189 · see citing works
Production and Titering of Recombinant Adeno-associated Viral Vectors
McClure, C., Marshall-Phelps, K. L., Wulff, P., Klugmann, M., & Murray, A. (2011) · Journal of Visualized Experiments · doi:10.3791/3348 · Cited by 137 · see citing works
CLIP-170 and IQGAP1 Cooperatively Regulate Dendrite Morphology
Swiech, L. J., Błażejczyk, M., Urbańska, M., Pietruszka, P., Dortland, B. R., Malik, A. R., Wulf, P. S., Hoogenraad, C. C., & Jaworski, J. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.6582-10.2011 · Cited by 107 · see citing works
Self-citationWood, L., Gray, N. W., Zhou, Z., Greenberg, M. E., & Shepherd, G. M. (2009) · Journal of Neuroscience · doi:10.1523/jneurosci.3321-09.2009 · Cited by 93 · see citing works
Rett Syndrome: Genes, Synapses, Circuits, and Therapeutics
Banerjee, A., Castro, J. E., & Sur, M. (2012) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2012.00034 · Cited by 65 · see citing works
Self-citation
Flagged first, then by citations
1 of the 43 references couldn’t be matched to a known paper.
Cited by
Sorted by citations
Single-cell transcriptomic analysis of Alzheimer’s disease
Mathys, H., Davila-Velderrain, J., Peng, Z., Gao, F., Mohammadi, S., Young, J. Z., Menon, M., He, L., Abdurrob, F., Jiang, X., et al. (2019) · Nature · doi:10.1038/s41586-019-1195-2 · Cited by 2,568 · see citing works
In vivo genome editing using Staphylococcus aureus Cas9
Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687 · see citing works
Self-citationComparative Analysis of Single-Cell RNA Sequencing Methods
Ziegenhain, C., Vieth, B., Parekh, S., Reinius, B., Guillaumet-Adkins, A., Smets, M., Leonhardt, H., Heyn, H., Hellmann, I., & Enard, W. (2017) · Molecular Cell · doi:10.1016/j.molcel.2017.01.023 · Cited by 1,494 · see citing works
Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors
Konermann, S., Lotfy, P., Brideau, N. J., Oki, J., Shokhirev, M. N., & Hsu, P. D. (2018) · Cell · doi:10.1016/j.cell.2018.02.033 · Cited by 1,164 · see citing works
“7 kb) makes it challenging to effectively deliver many single effector CRISPR enzymes (Abudayyeh et al., 2017; Ran et al., 2015; Swiech et al., 2015).”
InfluentialHigh-throughput functional genomics using CRISPR-Cas9
Shalem, O., Sanjana, N. E., & Zhang, F. (2015) · Nature Reviews Genetics · doi:10.1038/nrg3899 · Cited by 1,233 · see citing works
Self-citationTherapeutic genome editing: prospects and challenges
Cox, D. B. T., Platt, R. J., & Zhang, F. (2015) · Nature Medicine · doi:10.1038/nm.3793 · Cited by 1,336 · see citing works
Self-citationDelivering CRISPR: a review of the challenges and approaches
Lino, C. A., Harper, J. C., Carney, J. P., & Timlin, J. A. (2018) · Drug Delivery · doi:10.1080/10717544.2018.1474964 · Cited by 1,185 · see citing works
“Many groups have reported success packaging the SpCas9 and sgRNA into two separate AAV particles and using them for co-infection (Swiech et al., 2015; Hung et al., 2016).”
BackgroundIn vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Nelson, C. E., Hakim, C. H., Ousterout, D. G., Thakore, P. I., Moreb, E. A., Rivera, R. M. C., Madhavan, S., Pan, X., Ran, F. A., Yan, W. X., et al. (2016) · Science · doi:10.1126/science.aad5143 · Cited by 1,144 · see citing works
Self-citationCRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
Komor, A. C., Badran, A. H., & Liu, D. R. (2016) · Cell · doi:10.1016/j.cell.2016.10.044 · Cited by 1,011 · see citing works
Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array
Zetsche, B., Heidenreich, M., Mohanraju, P., Fedorova, I., Kneppers, J., DeGennaro, E. M., Winblad, N., Choudhury, S. R., Abudayyeh, O. O., Gootenberg, J. S., et al. (2017) · Nature Biotechnology · doi:10.1038/nbt.3737 · Cited by 926 · see citing works
“This plasmid also included an green fluorescent protein (GFP) fused to the KASH nuclear transmembrane domain 4 to enable FACS of targeted cell nuclei 5 .”
MethodResultSelf-citationApplications of CRISPR technologies in research and beyond
Barrangou, R., & Doudna, J. A. (2016) · Nature Biotechnology · doi:10.1038/nbt.3659 · Cited by 1,005 · see citing works
Yin, H., Song, C.-Q., Dorkin, J. R., Zhu, L. J., Li, Y., Wu, Q., Park, A., Yang, J., Suresh, S., Bizhanova, A., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3471 · Cited by 876 · see citing works
Self-citationBeyond editing: repurposing CRISPR-Cas9 for precision genome regulation and interrogation
Dominguez, A. A., Lim, W. A., & Qi, L. S. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2015.2 · Cited by 832 · see citing works
A human brain vascular atlas reveals diverse mediators of Alzheimer’s risk
Yang, A. C., Vest, R. T., Kern, F., Lee, D. P., Agam, M., Maat, C. A., Losada, P. M., Chen, M. B., Schaum, N., Khoury, N., et al. (2022) · Nature · doi:10.1038/s41586-021-04369-3 · Cited by 797 · see citing works
Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
Zuo, E., Sun, Y., Wei, W., Yuan, T., Ying, W., Sun, H., Yuan, L., Steinmetz, L. M., Li, Y., & Yang, H. (2019) · Science · doi:10.1126/science.aav9973 · Cited by 782 · see citing works
Self-citationIn vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni
Kim, E., Koo, T., Park, S. W., Kim, D., Kim, K., Cho, H.-Y., Song, D. W., Lee, K. J., Jung, M. H., Kim, S., et al. (2017) · Nature Communications · doi:10.1038/ncomms14500 · Cited by 715 · see citing works
SHANK proteins: roles at the synapse and in autism spectrum disorder
Monteiro, P., & Feng, G. (2017) · Nature Reviews Neuroscience · doi:10.1038/nrn.2016.183 · Cited by 681 · see citing works
Engineered Virus-like Particles for Efficient in Vivo Delivery of Therapeutic Proteins
Banskota, S., Raguram, A., Suh, S., Du, S. W., Davis, J. R., Choi, E. H., Wang, X., Nielsen, S. C., Newby, G. A., Randolph, P. B., et al. (2022) · Cell · doi:10.1016/j.cell.2021.12.021 · Cited by 668 · see citing works
“We produced v4 BE-eVLPs that install a silent mutation in mouse Dnmt1, a genomic locus known to be amenable to nuclease-mediated indel formation and adenine base editing in vivo (Levy et al., 2020; Swiech et al., 2015).”
MethodA Multifunctional AAV-CRISPR-Cas9 and Its Host Response
Chew, W. L., Tabebordbar, M., Cheng, J. K. W., Mali, P., Wu, E. Y., Ng, A. H. M., Zhu, K., Wagers, A. J., & Church, G. M. (2016) · Nature Methods · doi:10.1038/nmeth.3993 · Cited by 617 · see citing works
Dysregulation of brain and choroid plexus cell types in severe COVID-19
Yang, A. C., Kern, F., Losada, P. M., Agam, M. R., Maat, C. A., Schmartz, G. P., Fehlmann, T., Stein, J. A., Schaum, N., Lee, D. P., et al. (2021) · Nature · doi:10.1038/s41586-021-03710-0 · Cited by 620 · see citing works
Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications
Liu, C., Zhang, L., Liu, H., & Cheng, K. (2017) · Journal of Controlled Release · doi:10.1016/j.jconrel.2017.09.012 · Cited by 607 · see citing works
“Swiech and colleagues used two separated AAVs to deliver SpCas9 and sgRNAs to disrupt a single gene (Mecp2) or multiple genes (Dnmt1, Dnmt3a and Dnmt3b) in the mouse brain via stereotactic injection [96].”
Div-Seq: Single-nucleus RNA-Seq reveals dynamics of rare adult newborn neurons
Habib, N., Li, Y., Heidenreich, M., Swiech, L., Avraham-Davidi, I., Trombetta, J. J., Hession, C., Zhang, F., & Regev, A. (2016) · Science · doi:10.1126/science.aad7038 · Cited by 601 · see citing works
Self-citationDelivery technologies for genome editing
Yin, H., Kauffman, K. J., & Anderson, D. G. (2017) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.280 · Cited by 597 · see citing works
Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00799 · Cited by 560 · see citing works
Programmable RNA Tracking in Live Cells with CRISPR/Cas9
Nelles, D. A., Fang, M. Y., O’Connell, M. R., Xu, J. L., Markmiller, S., Doudna, J. A., & Yeo, G. W.-M. (2016) · Cell · doi:10.1016/j.cell.2016.02.054 · Cited by 526 · see citing works
“Efforts toward Cas9 delivery in vivo are underway (Dow et al., 2015; Swiech et al., 2015; Zuris et al., 2015), and these efforts combined with existing oligonucleotide chemistries (Bennett and Swayze, 2010) could support in vivo delivery of the RCas9 system for targetedmodulation of many features…”
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
Yin, H., Song, C.-Q., Suresh, S., Wu, Q., Walsh, S., Rhym, L. H., Mintzer, E., Bolukbasi, M. F., Zhu, L. J., Kauffman, K., et al. (2017) · Nature Biotechnology · doi:10.1038/nbt.4005 · Cited by 499 · see citing works
Levy, J. M., Yeh, W.-H., Pendse, N. D., Davis, J. R., Hennessey, E., Butcher, R. M., Koblan, L. W., Comander, J., Liu, Q., & Liu, D. R. (2020) · Nature Biomedical Engineering · doi:10.1038/s41551-019-0501-5 · Cited by 472 · see citing works
Poly(ADP-ribose) drives pathologic α-synuclein neurodegeneration in Parkinson’s disease
Kam, T.-I., Mao, X., Park, H., Chou, S.-C., Karuppagounder, S. S., Umanah, G. E., Yun, S. P., Brahmachari, S., Panicker, N., Chen, R., et al. (2018) · Science · doi:10.1126/science.aat8407 · Cited by 476 · see citing works
“S2A) (11) or used cortical cultures from PARP-1 knockouts (KOs) (Fig.”
BackgroundBase editing: advances and therapeutic opportunities
Porto, E. M., Komor, A. C., Slaymaker, I. M., & Yeo, G. W.-M. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0084-6 · Cited by 475 · see citing works
Ousterout, D. G., Kabadi, A. M., Thakore, P. I., Majoros, W. H., Reddy, T. E., & Gersbach, C. A. (2015) · Nature Communications · doi:10.1038/ncomms7244 · Cited by 470 · see citing works
“Immortalized myoblasts 70 from a DMD patient harboring a deletion of exons 48–50 (Δ48–50) in the dystrophin gene were maintained in skeletal muscle media (PromoCell) supplemented with 20% fetal bovine calf serum (Sigma), 50 μg/ml fetuin, 10 ng/ml human epidermal growth factor (Sigma), 1 ng/ml human…”
In vivo target gene activation via CRISPR/Cas9-mediated trans-epigenetic modulation
Liao, H.-K., Hatanaka, F., Araoka, T., Reddy, P., Wu, M.-Z., Sui, Y., Yamauchi, T., Sakurai, M., O’Keefe, D. D., Núñez-Delicado, E., et al. (2017) · Cell · doi:10.1016/j.cell.2017.10.025 · Cited by 451 · see citing works
“…2015 Addgene Plasmid#63798 MS2-P65-HSF1_GFP Konermann et al., 2015 Addgene Plasmid#61423 M-SPn-VP64 Esvelt et al., 2013 Addgene Plasmid#48674 PX551 Swiech et al., 2015 Addgene Plasmid#60957 AAV-tLuc This paper N/A AAV-tLuc-mCherry This paper N/A U6-dgRNA-CAG-MPH This paper…”
High levels of AAV vector integration into CRISPR-induced DNA breaks
Hanlon, K. S., Kleinstiver, B. P., Garcia, S. P., Zaborowski, M. P., Volak, A., Spirig, S. E., Muller, A., Sousa, A. A., Tsai, S. Q., Bengtsson, N. E., et al. (2019) · Nature Communications · doi:10.1038/s41467-019-12449-2 · Cited by 431 · see citing works
Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
Wang, S.-W., Gao, C., Zheng, Y.-M., Yi, L., Lu, J.-C., Huang, X.-Y., Cai, J.-B., Zhang, P.-F., Cui, Y.-H., & Ke, A.-W. (2022) · Molecular Cancer · doi:10.1186/s12943-022-01518-8 · Cited by 478 · see citing works
“The AAV dual-vector system successfully targets a single gene or multiple genes in the mouse brain and characterizes the influences of genome modification on neurons [129], suggesting that that AAV-mediated genome editing can be applied to study brain gene function.”
BackgroundSelf-citationCRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease
Yang, S., Chang, R., Yang, H., Zhao, T., Hong, Y., Kong, H. E., Sun, X., Qin, Z. S., Jin, P., Li, S., et al. (2017) · Journal of Clinical Investigation · doi:10.1172/jci92087 · Cited by 400 · see citing works
“Two gRNAs (T1 and T3) are expressed under the U6 promoter in an adeno-associated virus (AAV) vector that also expresses red fluorescent protein (RFP) (AAV-HTT-gRNA), and Cas9 is expressed in another AAV vector under the CMV promoter (AAV-CMV-Cas9) (13).”
BackgroundLee, B., Lee, K., Panda, S., Gonzales-Rojas, R., Chong, A., Bugay, V., Park, H. M., Brenner, R., Murthy, N., & Lee, H. Y. (2018) · Nature Biomedical Engineering · doi:10.1038/s41551-018-0252-8 · Cited by 361 · see citing works
“Currently, gene editing in the adult brain is mainly accomplished through the viral delivery of CRISPR-Cas9 6 .”
BackgroundInfluentialIn vivo CRISPR editing with no detectable genome-wide off-target mutations
Akçakaya, P., Bobbin, M. L., Guo, J. A., Malagon-Lopez, J., Clement, K., Garcia, S. P., Fellows, M. D., Porritt, M. J., Firth, M. A., Carreras, A., et al. (2018) · Nature · doi:10.1038/s41586-018-0500-9 · Cited by 316 · see citing works
Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works
CRISPR/Cas9-based engineering of the epigenome
Pulecio, J., Verma, N., Mejía-Ramírez, E., Huangfu, D., & Raya, A. (2017) · Cell Stem Cell · doi:10.1016/j.stem.2017.09.006 · Cited by 300 · see citing works
“Cas9 packaged into adeno-associated viruses (AAVs) has previously been used for in vivo genome editing of muscle, liver, and brain tissues to either produce desired mutations or to correct disease causing mutations (Swiech et al., 2015; Long et al., 2016; Nelson et al., 2016; Tabebordbar et al., 2016; Yang et al., 201…”
Chen, Z., Liu, F., Chen, Y., Liu, J., Wang, X., Chen, A. T., Deng, G., Zhang, H., Liu, J., Hong, Z., et al. (2017) · Advanced Functional Materials · doi:10.1002/adfm.201703036 · Cited by 297 · see citing works
In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models
Yin, C., Zhang, T., Qu, X., Zhang, Y., Putatunda, R., Xiao, X., Li, F., Xiao, W., Zhao, H., Dai, S., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.012 · Cited by 296 · see citing works
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
Li, B., Manan, R. S., Liang, S., Gordon, A. G. R., Jiang, A. Y., Varley, A., Gao, G., Langer, R., Xue, W., & Anderson, D. G. (2023) · Nature Biotechnology · doi:10.1038/s41587-023-01679-x · Cited by 291 · see citing works
DNA Breaks and End Resection Measured Genome-wide by End Sequencing
Canela, A., Sridharan, S., Sciascia, N., Tubbs, A., Meltzer, P., Sleckman, B. P., & Nussenzweig, A. (2016) · Molecular Cell · doi:10.1016/j.molcel.2016.06.034 · Cited by 284 · see citing works
“Applications Recently, CRISPR-associated Cas9 nuclease has been used to edit disease genes in adult mice (Swiech et al., 2015; Xue et al., 2014), and ZFNs have been utilized for gene therapy in HIV infection in humans (Tebas et al.”
Applications of CRISPR–Cas systems in neuroscience
Heidenreich, M., & Zhang, F. (2015) · Nature reviews. Neuroscience · doi:10.1038/nrn.2015.2 · Cited by 282 · see citing works
Self-citationCRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
Ruan, G.-X., Barry, E., Yu, D., Lukason, M., Cheng, S. H., & Scaria, A. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2016.12.006 · Cited by 270 · see citing works
Machine Learning in Genomic Medicine: A Review of Computational Problems and Data Sets
Leung, M. K. K., Delong, A., Alipanahi, B., & Frey, B. J. (2015) · Proceedings of the IEEE · doi:10.1109/jproc.2015.2494198 · Cited by 276 · see citing works
“The system has been used to control gene expression by deactivating the endonuclease domain [201], study gene functions in neural cells [202], develop synthetic biology applications [203], fix pathogenic mutations causing -thalassemia [204], and to repair cystic fibrosis transmembrane conductor receptor mutations [205…”
MethodPark, H., Oh, J., Shim, G., Cho, B., Chang, Y., Kim, S., Baek, S., Kim, H., Shin, J., Choi, H. G., et al. (2019) · Nature Neuroscience · doi:10.1038/s41593-019-0352-0 · Cited by 274 · see citing works
Viral Delivery Systems for CRISPR
Xu, C. L., Ruan, M. Z. C., Mahajan, V. B., & Tsang, S. H. (2019) · Viruses · doi:10.3390/v11010028 · Cited by 277 · see citing works
“used a dual-vector system to target Mecp2 in vivo in the adult mouse brain [26].”
MethodAstrocytes Regulate Daily Rhythms in the Suprachiasmatic Nucleus and Behavior
Tso, C. F., Simon, T., Greenlaw, A. C., Puri, T. A., Mieda, M., & Herzog, E. D. (2017) · Current Biology · doi:10.1016/j.cub.2017.02.037 · Cited by 262 · see citing works
“Loss of Daily Rhythms in SCN Astrocytes Lengthens Circadian Period in the SCN and in Behavior Using a recently developed gene editing technique [13, 14], we ablated the Bmal1 gene from targeted cells.”
Detecting and Avoiding Problems When Using the Cre/lox System
Song, A. J., & Palmiter, R. (2018) · Trends in Genetics · doi:10.1016/j.tig.2017.12.008 · Cited by 227 · see citing works
Artificial intelligence for precision medicine in neurodevelopmental disorders
Uddin, M. J., Wang, Y., & Woodbury‐Smith, M. (2019) · npj Digital Medicine · doi:10.1038/s41746-019-0191-0 · Cited by 257 · see citing works
Rett syndrome: insights into genetic, molecular and circuit mechanisms
Ip, J. P. K., Mellios, N., & Sur, M. (2018) · Nature reviews. Neuroscience · doi:10.1038/s41583-018-0006-3 · Cited by 247 · see citing works
Self-citationErratum: Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works
Potential pitfalls of CRISPR /Cas9‐mediated genome editing
Peng, R., Lin, G., & Li, J. (2015) · FEBS Journal · doi:10.1111/febs.13586 · Cited by 248 · see citing works
Colasante, G., Lignani, G., Brusco, S., Berardino, C. D., Carpenter, J. C., Giannelli, S. G., Valassina, N., Bido, S., Ricci, R., Castoldi, V., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.08.018 · Cited by 236 · see citing works
Clinical development of gene therapy: results and lessons from recent successes
Kumar, S. R. P., Markusic, D. M., Biswas, M., High, K. A., & Herzog, R. W. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.34 · Cited by 235 · see citing works
Efficient prime editing in mouse brain, liver and heart with dual AAVs
Davis, J. R., Banskota, S., Levy, J. M., Newby, G. A., Wang, X., Anzalone, A. V., Nelson, A. T., Chen, P. J., Hennes, A. D., An, M., et al. (2023) · Nature Biotechnology · doi:10.1038/s41587-023-01758-z · Cited by 237 · see citing works
“To enable the analysis of transduced cells, in addition to bulk tissue, we co-injected a 1 × 10 10 vg AAV9 expressing EGFP fused to a nuclear membrane-localized Klarsicht/ANC-1/Syne-1 homology (KASH) domain 32,42 (Fig.”
Zou, Y., Sun, X., Yang, Q., Zheng, M., Shimoni, O., Ruan, W., Wang, Y., Zhang, D., Yin, J., Huang, X., et al. (2022) · Science Advances · doi:10.1126/sciadv.abm8011 · Cited by 227 · see citing works
“Although the technology has already been used for brain diseases via intrabrain injections (3, 46), further clinical applications of gene therapy in brain diseases has been hampered by the lack of noninvasive, effective, and safe brain delivery systems for transporting CRISPR-Cas9 across the BBB to target diseased cel…”
MethodNishiyama, J., Mikuni, T., & Yasuda, R. (2017) · Neuron · doi:10.1016/j.neuron.2017.10.004 · Cited by 215 · see citing works
Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles
Luo, Y., Xu, C., Li, H., Cao, Z., Liu, J., Wang, J., Du, X., Yang, X., Gu, Z., & Wang, J. (2018) · ACS Nano · doi:10.1021/acsnano.7b07874 · Cited by 223 · see citing works
Mikuni, T., Nishiyama, J., Sun, Y., Kamasawa, N., & Yasuda, R. (2016) · Cell · doi:10.1016/j.cell.2016.04.044 · Cited by 214 · see citing works
Bioethical issues in genome editing by CRISPR-Cas9 technology
Ayanoğlu, F. B., ELÇİN, A. E., & Elçin, Y. M. (2020) · TURKISH JOURNAL OF BIOLOGY · doi:10.3906/biy-1912-52 · Cited by 233 · see citing works
“A mixture of green fluorescent protein (GFP)-labeled AAV-spCas9 and AAV-spGuide plasmids was transferred in vivo to the hippocampal toothed brain folds of adult male mice (Swiech et al., 2015).”
BackgroundCRISPR-Cas: New Tools for Genetic Manipulations from Bacterial Immunity Systems
Jiang, W., & Marraffini, L. A. (2015) · Annual Review of Microbiology · doi:10.1146/annurev-micro-091014-104441 · Cited by 210 · see citing works
Epigenome Editing: State of the Art, Concepts, and Perspectives
Kungulovski, G., & Jeltsch, A. (2015) · Trends in Genetics · doi:10.1016/j.tig.2015.12.001 · Cited by 208 · see citing works
“While our current mechanistic understanding is still very limited, in the future epigenome diting may become a therapeutic option in cases of psychiatric disease, disturbed behavior, and addiction [39,91,92].”
BackgroundEngineered materials for in vivo delivery of genome-editing machinery
Tong, S., Moyo, B., Lee, C. M., Leong, K. W., & Bao, G. (2019) · Nature Reviews Materials · doi:10.1038/s41578-019-0145-9 · Cited by 206 · see citing works
CRISPR Cpf1 proteins: structure, function and implications for genome editing
Safari, F., Zare, K., Negahdaripour, M., Barekati‐Mowahed, M., & Ghasemi, Y. (2019) · Cell & Bioscience · doi:10.1186/s13578-019-0298-7 · Cited by 196 · see citing works
“The first efforts in the Cas9-based multiplexing were performed in bacteria [65] and mammalian cells [66] by the simultaneous assembly of Cas9 complexes with different sgRNAs (crRNA fused to tracrRNA).”
BackgroundGyörgy, B., Lööv, C., Zaborowski, M. P., Takeda, S., Kleinstiver, B. P., Commins, C., Kastanenka, K. V., Mu, D., Volak, A., Giedraitis, V., et al. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.03.007 · Cited by 200 · see citing works
“60597 and 60958, respectively).(30) gRNA coding sequences were cloned into pX552 using SapI (Thermo Scientific, Waltham, MA).”
MethodViral vectors for therapy of neurologic diseases
Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P. (2017) · Neuropharmacology · doi:10.1016/j.neuropharm.2016.02.013 · Cited by 190 · see citing works
“AAVmediated genome editing using CRISPR-Cas9 has been shown to be effective in liver (Ran et al., 2015; Yang et al., 2016), dystrophic muscle (Tabebordbar et al., 2016; Nelson et al., 2016), and more crucially in the context of this review, in brain (Swiech et al., 2015).”
BackgroundInfluentialDelivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy
Gori, J. L., Hsu, P. D., Maeder, M. L., Shen, S., Welstead, G. G., & Bumcrot, D. A. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.074 · Cited by 192 · see citing works
“achieved robust multiplex gene modification at three genomic loci following administration into the mouse brain with this approach.(58) A novel approach to fit S.”
Viral Strategies for Targeting the Central and Peripheral Nervous Systems
Bedbrook, C. N., Deverman, B. E., & Gradinaru, V. (2018) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-080317-062048 · Cited by 168 · see citing works
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Gaj, T., Ojala, D. S., Ekman, F. K., Byrne, L. C., Limsirichai, P., & Schaffer, D. V. (2017) · Science Advances · doi:10.1126/sciadv.aar3952 · Cited by 185 · see citing works
“These results build on basic research studies by our laboratory (52) and others (53, 54) showing that CRISPR systems can induce gene editing in the mammalian brain and demonstrates that therapeutic genome editing can be achieved in tissues beyond the liver (55–58) and muscle (59–62) in animal models of human disease.”
ResultNGS Technologies as a Turning Point in Rare Disease Research , Diagnosis and Treatment
Marmiesse, A. F., Gouveia, S., & Couce, M. L. (2017) · Current Medicinal Chemistry · doi:10.2174/0929867324666170718101946 · Cited by 180 · see citing works
“to correct a hereditary disease, tyrosinemia, in a mouse model [230, 231].”
BackgroundRNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors
Thakore, P. I., Kwon, J. B., Nelson, C. E., Rouse, D. C., Gemberling, M. P., Oliver, M. L., & Gersbach, C. A. (2018) · Nature Communications · doi:10.1038/s41467-018-04048-4 · Cited by 173 · see citing works
Recent Progress in CRISPR/Cas9 Technology
Mei, Y., Wang, Y., Chen, H., Sun, Z. S., & Ju, X. (2016) · Journal of genetics and genomics/Journal of Genetics and Genomics · doi:10.1016/j.jgg.2016.01.001 · Cited by 181 · see citing works
“…Pten, p53 Cre-LoxP technology, hydrodynamic injection Xue et al., 2014 Brain Mecp2, Dnmt1, Dnmt3a, Dnmt3b AAV, hydrodynamic injection Swiech et al., 2015 In vitro Human hepatoma cell lines HBV cccDNA Transfection Dong et al., 2015 EPSC Tet1, Tet2, Tet3 Microinjection Wang et…”
MethodThe importance of DNA methylation of exons on alternative splicing
Shayevitch, R., Askayo, D., Keydar, I., & Ast, G. (2018) · RNA · doi:10.1261/rna.064865.117 · Cited by 171 · see citing works
Non-muscle myosin II in disease: mechanisms and therapeutic opportunities
Litwa, K., Horwitz, R., & Lamers, M. L. (2015) · Disease Models & Mechanisms · doi:10.1242/dmm.022103 · Cited by 167 · see citing works
“Furthermore, in genetically complex disorders, such as autism and schizophrenia, studies are needed to elucidate the contribution of specificCNV-associated genes,which is now possible through the use of gene editing technologies inmodel organisms (Swiech et al., 2014).”
BackgroundRewiring Neuronal Glycerolipid Metabolism Determines the Extent of Axon Regeneration
Yang, C., Wang, X., Wang, J., Wang, X., Chen, W., Lu, N., Siniossoglou, S., Yao, Z., & Liu, K. (2019) · Neuron · doi:10.1016/j.neuron.2019.10.009 · Cited by 170 · see citing works
“SV40 Penn Vector Cat#P1867 pAAV-U6. sgRNA(SapI)_hSyn-GFP-KASH-bGH Swiech et al., 2015 Cat#60958; RRID:Addgene_60958 Chemicals, Peptides, and Recombinant Proteins Cholera Toxin B Subunit, FITC Conjugate Sigma-Aldrich Cat#C1655 Fluorogold Invitrogen Cat#H22845 Papain Sigma-Aldrich…”
Lafora disease — from pathogenesis to treatment strategies
Nitschké, F., Ahonen, S., Nitschke, S., Mitra, S., & Minassian, B. A. (2018) · Nature Reviews Neurology · doi:10.1038/s41582-018-0057-0 · Cited by 169 · see citing works
Lau, C.-H., & Suh, Y. (2017) · F1000Research · doi:10.12688/f1000research.11243.1 · Cited by 161 · see citing works
Simultaneous repression of multiple bacterial genes using nonrepetitive extra-long sgRNA arrays
Reis, A. C., Halper, S. M., Vezeau, G. E., Cetnar, D. P., Hossain, A., Clauer, P. R., & Salis, H. M. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0286-9 · Cited by 157 · see citing works
Patterns of CRISPR/Cas9 activity in plants, animals and microbes
Bortesi, L., Zhu, C., Zischewski, J., Pérez, L., Bassié, L., Nadi, R., Forni, G., Lade, S. B., Soto, E., Jin, X., et al. (2016) · Plant Biotechnology Journal · doi:10.1111/pbi.12634 · Cited by 159 · see citing works
“Unlike plants, the CRISPR/Cas9 system can be used for genome editing in particular tissues by hydrodynamic injection or by introducing the components using Adeno-associated virus (AAV) or Adenovirus vectors (Rodriguez et al., 2014; Senis et al., 2014; Swiech et al., 2015; Xue et al., 2014).”
MethodEkman, F. K., Ojala, D. S., Adil, M. M., Zapana, P. A. L., Schaffer, D. V., & Gaj, T. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.07.009 · Cited by 163 · see citing works
CRISPR interference-based specific and efficient gene inactivation in the brain
Zheng, Y., Shen, W., Zhang, J., Yang, B., Liu, Y., Qi, H., Yu, X., Lu, S., Chen, Y., Xu, Y., et al. (2018) · Nature Neuroscience · doi:10.1038/s41593-018-0077-5 · Cited by 157 · see citing works
Self-citationSystematic analysis of CRISPR–Cas9 mismatch tolerance reveals low levels of off-target activity
Anderson, E. M., Haupt, A., Schiel, J. A., Chou, E. T., Machado, H. B., Strezoska, Ž., Lenger, S., McClelland, S., Birmingham, A., Vermeulen, A., et al. (2015) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2015.06.427 · Cited by 154 · see citing works
Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges
Luther, D. C., Lee, Y. W., Nagaraj, H., Scaletti, F., & Rotello, V. M. (2018) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2018.1517746 · Cited by 156 · see citing works
Multiplex Conditional Mutagenesis Using Transgenic Expression of Cas9 and sgRNAs
Yin, L., Maddison, L. A., Li, M., Kara, N., LaFave, M. C., Varshney, G. K., Burgess, S. M., Patton, J. G., & Chen, W. (2015) · Genetics · doi:10.1534/genetics.115.176917 · Cited by 153 · see citing works
“A Cas9 knock-in mouse line has been used to achieve tissue-specific mutagenesis (Platt et al. 2014; Swiech et al. 2015).”
MethodEngineered Viruses as Genome Editing Devices
Chen, X., & Gonçalves, M. A. F. V. (2015) · Molecular Therapy · doi:10.1038/mt.2015.164 · Cited by 151 · see citing works
Spatial control of in vivo CRISPR–Cas9 genome editing via nanomagnets
Zhu, H., Zhang, L., Tong, S., Lee, C. M., Deshmukh, H., & Bao, G. (2018) · Nature Biomedical Engineering · doi:10.1038/s41551-018-0318-7 · Cited by 151 · see citing works
Nucleus of the Solitary Tract Serotonin 5-HT2C Receptors Modulate Food Intake
D'Agostino, G., Lyons, D., Cristiano, C., Lettieri, M., Olarte-Sanchez, C., Burke, L. K., Greenwald-Yarnell, M., Cansell, C., Doslikova, B., Georgescu, T., et al. (2018) · Cell Metabolism · doi:10.1016/j.cmet.2018.07.017 · Cited by 149 · see citing works
“Two active sgRNAs were cloned into an AAV vector that also expressed eGFP to enable site-specific brain delivery (Swiech et al., 2015).”
MethodExosome-associated AAV vector as a robust and convenient neuroscience tool
Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy · doi:10.1038/gt.2016.11 · Cited by 146 · see citing works
“…various neuronal sub-population in vivo (12, 14), address functional connectivity between neurons and astrocytes (11), determine gene function using CRISPR-Cas9 (9, 10), and mediate spatial and temporal control of transgene expression in transgenic mice using the Cre-loxP approach (16).”
Programmable base editing of mutated TERT promoter inhibits brain tumour growth
Li, X., Qian, X., Wang, B., Xia, Y., Zheng, Y., Du, L., Xu, D., Xing, D., DePinho, R. A., & Lu, Z. (2020) · Nature Cell Biology · doi:10.1038/s41556-020-0471-6 · Cited by 151 · see citing works
Rapid and efficient one-step generation of paired gRNA CRISPR-Cas9 libraries
Vidigal, J. A., & Ventura, A. (2015) · Nature Communications · doi:10.1038/ncomms9083 · Cited by 147 · see citing works
Immunity to CRISPR Cas9 and Cas12a therapeutics
Chew, W. L. (2017) · WIREs Systems Biology and Medicine · doi:10.1002/wsbm.1408 · Cited by 146 · see citing works
Opportunities and challenges in modeling human brain disorders in transgenic primates
Jennings, C. G. B., Landman, R., Zhou, Y., Sharma, J., Hyman, J. B., Movshon, J. A., Qiu, Z., Roberts, A., Roe, A. W., Wang, X., et al. (2016) · Nature Neuroscience · doi:10.1038/nn.4362 · Cited by 140 · see citing works
Self-citationDirected evolution improves the catalytic efficiency of TEV protease
Sánchez, M. I., & Ting, A. Y. (2019) · Nature Methods · doi:10.1038/s41592-019-0665-7 · Cited by 143 · see citing works
Genome Editing Abrogates Angiogenesis in Vivo
Huang, X., Zhou, G., Wu, W., Duan, Y., Ma, G., Song, J., Xiao, R., Vandenberghe, L., Zhang, F., D’Amore, P. A., et al. (2017) · Nature Communications · doi:10.1038/s41467-017-00140-3 · Cited by 142 · see citing works
Self-citationTransgenic mice for in vivo epigenome editing with CRISPR-based systems
Gemberling, M. P., Siklenka, K., Rodriguez, E., Eisinger, K. R. T., Barrera, A., Liu, F., Kantor, A., Li, L., Cigliola, V., Hazlett, M. F., et al. (2021) · Nature Methods · doi:10.1038/s41592-021-01207-2 · Cited by 142 · see citing works
“28 This mouse line has been essential to numerous important breakthroughs including in vivo dissection of gene function 57 studies of gene regulatory networks, 58 and in vivo genetic screens.”
Base editing rescue of spinal muscular atrophy in cells and in mice
Arbab, M., Matuszek, Ż., Kray, K. M., Du, A., Newby, G. A., Blatnik, A. J., Raguram, A., Richter, M. F., Zhao, K. T., Levy, J. M., et al. (2023) · Science · doi:10.1126/science.adg6518 · Cited by 140 · see citing works
“GFP-positive cells as previously described(74, 76).”
Zhao, X., Liu, L., Lang, J., Cheng, K., Wang, Y., Li, X., Shi, J. Y., Wang, Y., & Nie, G. (2018) · Cancer Letters · doi:10.1016/j.canlet.2018.05.042 · Cited by 136 · see citing works
Surface-Engineered Viral Vectors for Selective and Cell Type-Specific Gene Delivery
Buchholz, C. J., Friedel, T., & Büning, H. (2015) · Trends in biotechnology · doi:10.1016/j.tibtech.2015.09.008 · Cited by 134 · see citing works
“Packaging the required components into viral vectors, local gene knockouts can be achieved by in vivo gene delivery [45].”
BackgroundGuo, Y., VanDusen, N. J., Zhang, L., Gu, W., Sethi, I., Guatimosim, S., Ma, Q., Jardin, B. D., Ai, Y., Zhang, D., et al. (2017) · Circulation Research · doi:10.1161/circresaha.116.310283 · Cited by 130 · see citing works
- Authors
- Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., Sur, M., & Zhang, F.
- Affiliations
- Broad Institute (US)
- Massachusetts Institute of Technology (US)
- Corresponding author
- Zhang, Feng
- Venue
- Nature Biotechnology
- Published
- 2014-10-19
- Volume
- 33
- Issue
- 1
- Pages
- 102-106
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC4492112
- OpenAlex
- W2056512830
- Cited by
- 772
- References
- 43
- Citations by year
- 2026: 17 · 2025: 32 · 2024: 47 · 2023: 56 · 2022: 47 · 2021: 66 · 2020: 76 · 2019: 85 · 2018: 102 · 2017: 90 · 2016: 87 · 2015: 65
- FWCI
- 33.10
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- DSpace@MIT (Massachusetts Institute of Technology) (submittedVersion, cc-by-nc)
- PMC
- Funders
- National Institute of Mental Health (DP1MH100706, R01MH085802)
- National Eye Institute (R01EY007023)
- National Institute of Neurological Disorders and Stroke (R01NS073124, 5R01-NS073124)
- National Center for Chronic Disease Prevention and Health Promotion (5DP1-MH100706)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Keywords
- CRISPR/Cas9, adeno-associated virus vectors, in vivo gene editing, MeCP2, DNMT1, DNMT3A, DNMT3B, DNA methylation, postmitotic neurons, adult mouse brain, gene function, guide RNA, electrophysiology, behavior
- MeSH terms
- Animals, Streptococcus pyogenes, Clustered Regularly Interspaced Short Palindromic Repeats
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
