In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
Open accessAbstract
Targeted genome editing via engineered nucleases is an exciting area of biomedical research and holds potential for clinical applications. Despite rapid advances in the field, in vivo targeted transgene integration is still infeasible because current tools are inefficient, especially for non-dividing cells, which compose most adult tissues. This poses a barrier for uncovering fundamental biological principles and developing treatments for a broad range of genetic disorders. Based on clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) technology, here we devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of postnatal mammals). As a proof of concept of its therapeutic potential, we demonstrate the efficacy of HITI in improving visual function using a rat model of the retinal degeneration condition retinitis pigmentosa. The HITI method presented here establishes new avenues for basic research and targeted gene therapies.
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?
Packaging CRISPR gRNA and Cas9 in AAV and delivering it subretinally can specifically disrupt a disease-causing allele in photoreceptors or RPE.
“By packaging the CRISPR gRNA and Cas9 enzyme-encoding transgene within AAV and delivering the gene editing vector into the subretinal space, it is possible to specifically disrupt the disease-causing allele in the photoreceptors or RPE [Suzuki 2016; Yu 2017; McCullough 2019].”
From Correcting visual loss by genetics and prosthetics (2020) · cites this paper as Suzuki 2016
HITI strategy allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and in vivo.
“This approach allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and in vivo [87].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [87]
Non-mitotic cells are very inefficient for HDR-based repair, which is main pathway to correct genetic disorders to date.
“Non-mitotic cells are very inefficient for HDR-based repair, which is the main pathway to correct genetic disorders to date [87, 200].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [87]
HITI keeps its high efficiency in non-mitotic cells, which are present in most adult tissues.
“More importantly, HITI keeps its high efficiency in the non-mitotic cells, which are present in most adult tissues [87].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [87]
HITI strategy exhibits much higher knock-in efficiency than HDR both in vitro and in vivo.
“The recently developed HITI strategy exhibits much higher knock-in efficiency than HDR both in vitro and in vivo [86, 87].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [87]
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
1 retracted
RETRACTED ARTICLE: A mechanism for the suppression of homologous recombination in G1 cells
Orthwein, A., Noordermeer, S. M., Wilson, M. D., Landry, S., Enchev, R. I., Sherker, A., Munro, M., Pinder, J., Salsman, J., Dellaire, G., et al. (2015) · Nature · doi:10.1038/nature16142 · Cited by 471 · see citing works
RetractedFast and accurate short read alignment with Burrows-Wheeler transform
Li, H., & Durbin, R. (2009) · Bioinformatics · doi:10.1093/bioinformatics/btp324 · Cited by 58,607 · 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
RNA-Guided Human Genome Engineering via Cas9
Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210 · see citing works
A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
Madisen, L., Zwingman, T. A., Sunkin, S. M., Oh, S. W., Zariwala, H. A., Gu, H., Ng, L. L., Palmiter, R. D., Hawrylycz, M. J., Jones, A. R., et al. (2010) · Nature Neuroscience · doi:10.1038/nn.2467 · Cited by 6,907 · see citing works
Millisecond-timescale, genetically targeted optical control of neural activity
Boyden, E. S., Zhang, F., Bamberg, E., Nagel, G., & Deisseroth, K. A. (2005) · Nature Neuroscience · doi:10.1038/nn1525 · Cited by 4,831 · 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
The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway
Lieber, M. R. (2010) · Annual Review of Biochemistry · doi:10.1146/annurev.biochem.052308.093131 · Cited by 2,882 · see citing works
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924 · see citing works
Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490 · see citing works
Therapeutic 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
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273 · see citing works
Gene therapy returns to centre stage
Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184 · see citing works
Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat
D'Cruz, P. M. (2000) · Human Molecular Genetics · doi:10.1093/hmg/9.4.645 · Cited by 869 · see citing works
Tabata, H., & Nakajima, K. (2001) · Neuroscience · doi:10.1016/s0306-4522(01)00016-1 · Cited by 705 · see citing works
Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors
Kutner, R. H., Zhang, X., & Reiser, J. (2009) · Nature Protocols · doi:10.1038/nprot.2009.22 · Cited by 670 · see citing works
Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair
Auer, T. O., Duroure, K., De Cian, A., Concordet, J.-P., & Del Bene, F. (2014) · Genome Research · doi:10.1101/gr.161638.113 · Cited by 631 · see citing works
A transcription activator-like effector toolbox for genome engineering
Sanjana, N. E., Cong, L., Zhou, Y., Cunniff, M. M., Feng, G., & Zhang, F. (2012) · Nature Protocols · doi:10.1038/nprot.2011.431 · Cited by 628 · see citing works
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 582 · see citing works
Recapitulation of premature ageing with iPSCs from Hutchinson-Gilford progeria syndrome
Liu, G.-H., Barkho, B. Z., Ruiz, S., Diep, D., Qu, J., Yang, S.-L., Panopoulos, A. D., Suzuki, K., Kurian, L., Walsh, C., et al. (2011) · Nature · doi:10.1038/nature09879 · Cited by 575 · see citing works
Self-citationTargeted genome editing in human repopulating haematopoietic stem cells
Genovese, P., Schiroli, G., Escobar, G., Di Tomaso, T., Firrito, C., Calabria, A., Moi, D., Mazzieri, R., Bonini, C., Holmes, M. C., et al. (2014) · Nature · doi:10.1038/nature13420 · Cited by 555 · see citing works
Nakade, S., Tsubota, T., Sakane, Y., Kume, S., Sakamoto, N., Obara, M., Daimon, T., Sezutsu, H., Yamamoto, T., Sakuma, T., et al. (2014) · Nature Communications · doi:10.1038/ncomms6560 · Cited by 493 · see citing works
Self-citationChen, Z.-Y., He, C.-Y., Ehrhardt, A., & Kay, M. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00168-0 · Cited by 463 · see citing works
Shitamukai, A., Konno, D., & Matsuzaki, F. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.4773-10.2011 · Cited by 450 · see citing works
Self-citationIs Non-Homologous End-Joining Really an Inherently Error-Prone Process?
Bétermier, M., Bertrand, P., & López, B. S. (2014) · PLoS Genetics · doi:10.1371/journal.pgen.1004086 · Cited by 420 · see citing works
Site-specific integration and tailoring of cassette design for sustainable gene transfer
Lombardo, A., Cesana, D., Genovese, P., Stefano, B. D., Provasi, E., Colombo, D., Neri, M., Magnani, Z. I., Cantore, A., Riso, P. L., et al. (2011) · Nature Methods · doi:10.1038/nmeth.1674 · Cited by 332 · see citing works
A robust system for production of minicircle DNA vectors
Kay, M. A., He, C.-Y., & Chen, Z.-Y. (2010) · Nature Biotechnology · doi:10.1038/nbt.1708 · Cited by 329 · see citing works
Maresca, M., Lin, V. G., Guo, N., & Yang, Y. (2012) · Genome Research · doi:10.1101/gr.145441.112 · Cited by 310 · see citing works
Belmonte, J. C. I., Callaway, E. M., Caddick, S. J., Churchland, P. S., Feng, G., Homanics, G. E., Lee, K., Leopold, D. A., Miller, C. T., Mitchell, J. F., et al. (2015) · Neuron · doi:10.1016/j.neuron.2015.03.021 · Cited by 269 · see citing works
Self-citationAn alternative pluripotent state confers interspecies chimaeric competency
Wu, J., Okamura, D., Li, M., Suzuki, K., Luo, C., Ma, L., He, Y., Li, Z., Benner, C. W., Tamura, I., et al. (2015) · Nature · doi:10.1038/nature14413 · Cited by 243 · see citing works
Self-citationTargeted Gene Correction of Laminopathy-Associated LMNA Mutations in Patient-Specific iPSCs
Liu, G., Suzuki, K., Qu, J. T., Sancho-Martinez, I., Yi, F., Li, M., Kumar, S., Nivet, E., Kim, J., Soligalla, R. D., et al. (2011) · Cell stem cell · doi:10.1016/j.stem.2011.04.019 · Cited by 219 · see citing works
Self-citationLinear DNAs Concatemerize in Vivo and Result in Sustained Transgene Expression in Mouse Liver
Chen, Z.-Y., Yant, S. R., He, C.-Y., Meuse, L. A., Shen, S., & Kay, M. A. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0278 · Cited by 186 · see citing works
Mizutani, K., & Saito, T. (2005) · Development · doi:10.1242/dev.01693 · Cited by 134 · see citing works
Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs
Liu, G.-H., Suzuki, K., Li, M., Qu, J. T., Montserrat, N., Tarantino, C., Gu, Y., Yi, F., Xu, X., Zhang, W., et al. (2014) · Nature Communications · doi:10.1038/ncomms5330 · Cited by 121 · see citing works
Self-citationHuman Retinal Progenitor Cell Transplantation Preserves Vision
Luo, J., Baranov, P. Y., Patel, S. H., Ouyang, H. W., Quach, J., Wu, F., Qiu, A., Luo, H., Hicks, C. A., Zeng, J., et al. (2014) · Journal of Biological Chemistry · doi:10.1074/jbc.m113.513713 · Cited by 113 · see citing works
Self-citationIsolation of Muscle Stem Cells by Fluorescence Activated Cell Sorting Cytometry
Pasut, A., Oleynik, P., & Rudnicki, M. A. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-343-1_3 · Cited by 111 · see citing works
Intravenous Injections in Neonatal Mice
Gombash, S. E., Kaspar, B. K., & Foust, K. D. (2014) · Journal of Visualized Experiments · doi:10.3791/52037 · Cited by 76 · see citing works
Isolation and analysis of single cells from the mouse heart
Pinto, A. R., Chandran, A. R., Rosenthal, N. A., & Godwin, J. W. (2013) · Journal of Immunological Methods · doi:10.1016/j.jim.2013.03.012 · Cited by 55 · see citing works
Sauvé, Y., Lu, B., & Lund, R. D. (2003) · Vision Research · doi:10.1016/j.visres.2003.08.015 · Cited by 51 · see citing works
The Intracellular Mobility of Nuclear Import Receptors and NLS Cargoes
Wu, J., Corbett, A. H., & Berland, K. M. (2009) · Biophysical Journal · doi:10.1016/j.bpj.2009.01.050 · Cited by 44 · see citing works
Self-citationRenal press-mediated transfection method for plasmid DNA and siRNA to the kidney
Mukai, H., Kawakami, S., & Hashida, M. (2008) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2008.04.097 · Cited by 40 · see citing works
Transferring genes into cultured mammalian embryos by electroporation
Takahashi, M. P., Nomura, T., & Osumi, N. (2008) · Development Growth & Differentiation · doi:10.1111/j.1440-169x.2008.01046.x · Cited by 39 · see citing works
Kameda, Y., Kameya, T., & Frankfurter, A. J. (1993) · Brain Research · doi:10.1016/0006-8993(93)90946-k · Cited by 22 · see citing works
Luchetti, A., Malgieri, A., & Sangiuolo, F. C. (2014) · Methods in molecular biology · doi:10.1007/978-1-62703-761-7_6 · Cited by 6 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works
Base editing: precision chemistry on the genome and transcriptome of living cells
Rees, H. A., & Liu, D. R. (2018) · Nature Reviews Genetics · doi:10.1038/s41576-018-0059-1 · Cited by 1,675 · see citing works
Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
Walton, R. T., Christie, K. A., Whittaker, M. N., & Kleinstiver, B. P. (2020) · Science · doi:10.1126/science.aba8853 · Cited by 1,233 · see citing works
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Charlesworth, C. T., Deshpande, P. S., Dever, D. P., Camarena, J., Lemgart, V. T., Cromer, M. K., Vakulskas, C. A., Collingwood, M. A., Zhang, L., Bode, N. M., et al. (2019) · Nature Medicine · doi:10.1038/s41591-018-0326-x · Cited by 1,093 · see citing works
Reprogramming human T cell function and specificity with non-viral genome targeting
Roth, T. L., Puig-Saus, C., Yu, R., Shifrut, E., Carnevale, J., Li, P. J., Hiatt, J., Saco, J., Krystofinski, P., Li, H., et al. (2018) · Nature · doi:10.1038/s41586-018-0326-5 · Cited by 839 · see citing works
Advances in the delivery of RNA therapeutics: from concept to clinical reality
Kaczmarek, J. C., Kowalski, P. S., & Anderson, D. G. (2017) · Genome Medicine · doi:10.1186/s13073-017-0450-0 · Cited by 656 · see citing works
Niche stiffness underlies the ageing of central nervous system progenitor cells
Segel, M., Neumann, B., Hill, M. F. E., Weber, I. P., Viscomi, C., Zhao, C., Young, A., Agley, C. C., Thompson, A. J., Gonzalez, G. A., et al. (2019) · Nature · doi:10.1038/s41586-019-1484-9 · Cited by 532 · see citing works
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
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
Self-citationNon-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities
Li, L., Hu, S., & Chen, X. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2018.04.031 · Cited by 415 · see citing works
Harnessing genomic information for livestock improvement
Georges, M., Charlier, C., & Hayes, B. (2019) · Nature Reviews Genetics · doi:10.1038/s41576-018-0082-2 · Cited by 384 · 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
Functional Genetic Variants Revealed by Massively Parallel Precise Genome Editing
Sharon, E., Chen, S.-A. A., Khosla, N. M., Smith, J. D., Pritchard, J. K., & Fraser, H. B. (2018) · Cell · doi:10.1016/j.cell.2018.08.057 · Cited by 225 · see citing works
Self-citationDelivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
Taha, E. A., Lee, J., & Hotta, A. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.01.013 · Cited by 259 · see citing works
A New Class of Medicines through DNA Editing
Porteus, M. H. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1800729 · Cited by 229 · see citing works
Early and late stage gene therapy interventions for inherited retinal degenerations
Botto, C., Rucli, M., Tekinsoy, M. D., Pulman, J., Sahel, J.-A., & Dalkara, D. (2022) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2021.100975 · Cited by 190 · see citing works
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
Riesenberg, S., & Maricic, T. (2018) · Nature Communications · doi:10.1038/s41467-018-04609-7 · Cited by 174 · see citing works
Schober, K., Müller, T. R., Gökmen, F., Grassmann, S., Effenberger, M., Poltorak, M., Stemberger, C., Schumann, K., Roth, T. L., Marson, A., et al. (2019) · Nature Biomedical Engineering · doi:10.1038/s41551-019-0409-0 · Cited by 178 · see citing works
Insulin-Degrading Enzyme in the Fight against Alzheimer's Disease
Kurochkin, I. V., Guarnera, E., & Berezovsky, I. N. (2018) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2017.10.008 · Cited by 185 · see citing works
Seeing the light after 25 Years of retinal gene therapy
Trapani, I., & Auricchio, A. (2018) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2018.06.006 · Cited by 124 · see citing works
Tsai, Y.-T., Wu, W.-H., Lee, T.-T., Wu, W.-P., Xu, C. L., Park, K. S., Cui, X., Justus, S., Lin, C.-S., Jauregui, R., et al. (2018) · Ophthalmology · doi:10.1016/j.ophtha.2018.04.001 · Cited by 119 · see citing works
Tools for efficient genome editing; ZFN, TALEN, and CRISPR
Shamshirgaran, Y., Liu, J., Sumer, H., Verma, P. J., & Taheri-Ghahfarokhi, A. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2301-5_2 · Cited by 114 · see citing works
Gene Therapy in Retinal Dystrophies
Ziccardi, L., Cordeddu, V., Gaddini, L., Matteucci, A., Parravano, M., Malchiodi-Albedi, F., & Varano, M. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20225722 · Cited by 107 · see citing works
Gaj, T., Staahl, B. T., Rodrigues, G. M. C., Limsirichai, P., Ekman, F. K., Doudna, J. A., & Schaffer, D. V. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx154 · Cited by 89 · see citing works
Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges
Vázquez-Domínguez, I., Garanto, A., & Collin, R. W. J. (2019) · Genes · doi:10.3390/genes10090654 · Cited by 80 · see citing works
Kumar, N., Stanford, W., de Solis, C., Aradhana, Abraham, N. D., Dao, T.-M. J., Thaseen, S., Sairavi, A., Gonzalez, C. U., & Ploski, J. E. (2018) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2018.00413 · Cited by 79 · see citing works
Visual prosthesis: interfacing stimulating electrodes with retinal neurons to restore vision.
Barriga-Rivera, A., Bareket, L., Goding, J., Aregueta-Robles, U. A., & Suaning, G. J. (2017) · Frontiers in Neuroscience · doi:10.3389/fnins.2017.00620 · Cited by 73 · see citing works
Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina
Trapani, I. (2019) · Genes · doi:10.3390/genes10040287 · Cited by 73 · see citing works
Recent advances in gene therapy for neurodevelopmental disorders with epilepsy
Turner, T. J., Zourray, C., Schorge, S., & Lignani, G. (2021) · Journal of Neurochemistry · doi:10.1111/jnc.15168 · Cited by 71 · see citing works
Dever, D. P., & Porteus, M. H. (2017) · Current Opinion in Hematology · doi:10.1097/moh.0000000000000385 · Cited by 62 · see citing works
The photoreceptor cilium and its diseases
Bachmann-Gagescu, R., & Neuhauss, S. C. (2019) · Current Opinion in Genetics & Development · doi:10.1016/j.gde.2019.05.004 · Cited by 60 · see citing works
Genome-wide detection of CRISPR editing in vivo using GUIDE-tag
Liang, S.-Q., Liu, P., Smith, J. L., Mintzer, E., Maitland, S., Dong, X., Yang, Q., Lee, J., Haynes, C. M., Zhu, L. J., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-28135-9 · Cited by 54 · see citing works
Jo, D. H., Koo, T., Cho, C. S., Kim, J. H., Kim, J.-S., & Kim, J. H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2018.10.009 · Cited by 57 · see citing works
Ou, L., Przybilla, M. J., Ahlat, O., Kim, S., Overn, P., Jarnes, J., O’Sullivan, M. G., & Whitley, C. B. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.03.018 · Cited by 56 · see citing works
Massively targeted evaluation of therapeutic CRISPR off-targets in cells
Pan, X., Qu, K., Yuan, H., Xiang, X., Anthon, C., Pashkova, L., Liang, X., Han, P., Corsi, G. I., Xu, F., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-31543-6 · Cited by 42 · see citing works
Genome-editing strategies for treating human retinal degenerations
Quinn, J., Musa, A., Kantor, A., McClements, M. E., Cehajic-Kapetanovic, J., MacLaren, R. E., & Xue, K. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.231 · Cited by 41 · see citing works
Directed evolution of hyperactive integrases for site specific insertion of transgenes
Hew, B. E., Gupta, S., Sato, R., Waller, D. F., Stoytchev, I., Short, J. E., Sharek, L., Tran, C. T., Badran, A. H., & Owens, J. B. (2024) · Nucleic Acids Research · doi:10.1093/nar/gkae534 · Cited by 35 · see citing works
Correcting visual loss by genetics and prosthetics
Xue, K., & MacLaren, R. E. (2020) · Current Opinion in Physiology · doi:10.1016/j.cophys.2020.03.003 · Cited by 5 · see citing works
- Authors
- Suzuki, K., Tsunekawa, Y., Hernandez-Benitez, R., Wu, J., Zhu, J., Kim, E. J., Hatanaka, F., Yamamoto, M., Araoka, T., Li, Z., Kurita, M., Hishida, T., Li, M., Aizawa, E., Guo, S., Chen, S., Goebl, A., Soligalla, R. D., Qu, J., Jiang, T., Fu, X., Jafari, M., Esteban, C. R., Berggren, W. T., Lajara, J., Nuñez-Delicado, E., Guillen, P., Campistol, J. M., Matsuzaki, F., Liu, G.-H., Magistretti, P., Zhang, K., Callaway, E. M., Zhang, K., & Belmonte, J. C. I.
- Affiliations
- Salk Institute for Biological Studies (US)
- RIKEN (JP)
- University of California San Diego (US)
- Chinese Academy of Sciences (CN)
- Consorci Institut D'Investigacions Biomediques August Pi I Sunyer (ES)
- Jinan University (CN)
- King Abdullah University of Science and Technology (SA)
- Sichuan University (CN)
- ORCID
- Suzuki, Keiichiro
- Tsunekawa, Yuji
- Wu, Jun
- Zhu, Jie
- Kim, Euiseok J.
- Hatanaka, Fumiyuki
- Araoka, Toshikazu
- Li, Zhe
- Kurita, Masakazu
- Hishida, Tomoaki
- Li, Mo
- Aizawa, Emi
- Guo, Shicheng
- Chen, Song
- Goebl, April
- Qu, Jing
- Fu, Xin
- Jafari, Maryam
- Campistol, Josep M.
- Matsuzaki, Fumio
- Liu, Guang-Hui
- Magistretti, Pierre
- Zhang, Kun
- Callaway, Edward M.
- Zhang, Kang
- Venue
- Nature
- Published
- 2016-11-16
- Volume
- 540
- Issue
- 7631
- Pages
- 144-149
- ISSN
- 0028-0836 (ISSN-L)
- 1476-4687
- PMC
- PMC5331785
- OpenAlex
- W2550079706
- Cited by
- 1,150
- References
- 45
- Citations by year
- 2026: 65 · 2025: 104 · 2024: 101 · 2023: 113 · 2022: 117 · 2021: 125 · 2020: 138 · 2019: 137 · 2018: 132 · 2017: 114 · 2016: 3 · 2015: 1
- FWCI
- 66.85
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Nature (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- RePEc: Research Papers in Economics (submittedVersion)
- King Abdullah University of Science and Technology Repository (King Abdullah University of Science and Technology) (submittedVersion)
- The Molecular Biology Society of Japan
- Funders
- National Heart, Lung, and Blood Institute (R01HL123755)
- National Eye Institute (P30EY019005, P30EY022589)
- National Cancer Institute (P30CA014195)
- National Institute of Neurological Disorders and Stroke (R24NS092943, P30NS047101)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- CRISPR/Cas9, non-homologous end joining, in vivo gene editing, postmitotic cells, neurons, retinitis pigmentosa, retinal degeneration, gene therapy, visual function
- MeSH terms
- Gene Editing, Animals, Cell Division, Disease Models, Animal, Neurons, Retinitis Pigmentosa, Genetic Therapy, Genome, Sequence Homology, Gene Targeting, Rats, Gene Knock-In Techniques, CRISPR-Cas Systems
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
