Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

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

Genome 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

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

The new frontier of genome engineering with CRISPR-Cas9

Doudna, J. A., & Charpentier, E. (2014) · Science · doi:10.1126/science.1258096 · Cited by 7,062 · see citing works

Development 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

Genome-scale CRISPR-Cas9 knockout screening in human cells

Shalem, O., Sanjana, N. E., Hartenian, E., Shi, X., Scott, D. A., Mikkelsen, T. S., Heckl, D., Ebert, B. L., Root, D. E., Doench, J. G., et al. (2014) · Science · doi:10.1126/science.1247005 · Cited by 5,234 · 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

Genetic screens in human cells using the CRISPR-Cas9 system

Wang, T., Wei, J. J., Sabatini, D. M., & Lander, E. S. (2014) · Science · doi:10.1126/science.1246981 · Cited by 2,856 · 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

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

Brouns, S. J. J., Jore, M. M., Lundgren, M., Westra, E. R., Slijkhuis, R. J. H., Snijders, A. P., Dickman, M. J., Makarova, K. S., Koonin, E. V., & John van der, O. O. S. T. (2008) · Science · doi:10.1126/science.1159689 · Cited by 2,593 · see citing works

CRISPR RNA Maturation by Trans-Encoded Small RNA and Host Factor RNase III

Deltcheva, E., Chylinski, K., Sharma, C. M., Gonzales, K., Chao, Y., Pirzada, Z. A., Eckert, M. R., Vogel, J., & Charpentier, E. (2011) · Nature · doi:10.1038/nature09886 · Cited by 2,563 · see citing works

Evolution and classification of the CRISPR–Cas systems

Makarova, K. S., Haft, D. H., Barrangou, R., Brouns, S. J. J., Charpentier, E., Horvath, P., Moineau, S., Mojica, F. J. M., Wolf, Y. I., Yakunin, A. F., et al. (2011) · Nature Reviews Microbiology · doi:10.1038/nrmicro2577 · Cited by 2,512 · see citing works

An updated evolutionary classification of CRISPR–Cas systems

Makarova, K. S., Wolf, Y. I., Alkhnbashi, O. S., Costa, F., Shah, S. A., Saunders, S. J., Barrangou, R., Brouns, S. J. J., Charpentier, E., Haft, D. H., et al. (2015) · Nature Reviews Microbiology · doi:10.1038/nrmicro3569 · Cited by 2,504 · see citing works

Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product

Ishino, Y., Shinagawa, H., Makino, K., Amemura, M., & Nakata, A. (1987) · Journal of Bacteriology · doi:10.1128/jb.169.12.5429-5433.1987 · Cited by 2,421 · see citing works

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

Garneau, J. E., Dupuis, M.-È., Villion, M., Romero, D. A., Barrangou, R., Boyaval, P., Fremaux, C., Horvath, P., Magadán, A. H., & Moineau, S. (2010) · Nature · doi:10.1038/nature09523 · Cited by 2,336 · see citing works

Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign Genetic Elements

Mojica, F. J. M., D�ez-Villase�or, C., Garc�a-Mart�nez, J., & Soria, E. (2005) · Journal of Molecular Evolution · doi:10.1007/s00239-004-0046-3 · Cited by 2,219 · see citing works

Identification of genes that are associated with DNA repeats in prokaryotes

Jansen, R., van Embden, J. D. A., Gaastra, W., & Schouls, L. M. (2002) · Molecular Microbiology · doi:10.1046/j.1365-2958.2002.02839.x · Cited by 2,035 · 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

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

Kim, S., Kim, D., Cho, S. W., Kim, J., & Kim, J.-S. (2014) · Genome Research · doi:10.1101/gr.171322.113 · Cited by 1,856 · see citing works

Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L., & Trono, D. (1998) · Journal of Virology · doi:10.1128/jvi.72.12.9873-9880.1998 · Cited by 1,815 · see citing works

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

Mali, P., Aach, J., Stranges, P. B., Esvelt, K. M., Moosburner, M., Kosuri, S., Yang, L., & Church, G. M. (2013) · Nature Biotechnology · doi:10.1038/nbt.2675 · Cited by 1,752 · cited 1× · see citing works

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA

Liu, F., Song, Y. K., & Liu, D. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300947 · Cited by 1,728 · see citing works

One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering

Yang, H., Wang, H., Shivalila, C. S., Cheng, A. W., Shi, L., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.08.022 · Cited by 1,596 · see citing works

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508 · see citing works

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

Zuris, J. A., Thompson, D. B., Shu, Y., Guilinger, J. P., Bessen, J. L., Hu, J. H., Maeder, M. L., Joung, J. K., Chen, Z.-Y., & Liu, D. R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3081 · Cited by 1,460 · see citing works

Gene Editing of <i>CCR5</i> in Autologous CD4 T Cells of Persons Infected with HIV

Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357 · see citing works

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

Koike-Yusa, H., Li, Y., Tan, E.-P., Velasco-Herrera, M. D. C., & Yusa, K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2800 · Cited by 1,064 · see citing works

Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

Niu, Y., Shen, B., Cui, Y., Chen, Y., Wang, J., Wang, L., Kang, Y., Zhao, X., Si, W., Li, W., et al. (2014) · Cell · doi:10.1016/j.cell.2014.01.027 · Cited by 1,033 · see citing works

Effect 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

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science · doi:10.1126/science.1233151 · Cited by 1,025 · see citing works

Safety and efficacy of RNAi therapy for transthyretin amyloidosis

Coelho, T., Adams, D., Silva, A., Lozeron, P., Hawkins, P. N., Mant, T., Perez, J., Chiesa, J., Warrington, S., Tranter, E., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1208760 · Cited by 971 · 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

Heritable genome editing in C. elegans via a CRISPR-Cas9 system

Friedland, A. E., Tzur, Y. B., Esvelt, K. M., Colaiácovo, M. P., Church, G. M., & Calarco, J. A. (2013) · Nature Methods · doi:10.1038/nmeth.2532 · Cited by 899 · see citing works

Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis

Chen, S., Sanjana, N. E., Zheng, K., Shalem, O., Lee, K., Shi, X., Scott, D. A., Song, J. W., Pan, J. Q., Weissleder, R., et al. (2015) · Cell · doi:10.1016/j.cell.2015.02.038 · Cited by 891 · see citing works

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

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

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

Shen, B., Zhang, W., Zhang, J., Zhou, J., Wang, J.-Y., Chen, L., Wang, L., Hodgkins, A., Iyer, V., Huang, X., et al. (2014) · Nature Methods · doi:10.1038/nmeth.2857 · Cited by 827 · see citing works

Genome editing with RNA-guided Cas9 nuclease in Zebrafish embryos

Chang, N., Sun, C., Gao, L., Zhu, D., Xu, X., Zhu, X., Xiong, J., & Xi, J. (2013) · Cell Research · doi:10.1038/cr.2013.45 · Cited by 819 · 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

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

Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., Sur, M., & Zhang, F. (2015) · Nature Biotechnology · doi:10.1038/nbt.3055 · Cited by 772 · cited 1× · see citing works

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

Liang, X., Potter, J., Kumar, S., Zou, Y., Quintanilla, R., Sridharan, M., Carte, J., Chen, W., Roark, N., Ranganathan, S., et al. (2015) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2015.04.024 · Cited by 733 · see citing works

High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells

Zhou, Y., Zhu, S., Cai, C., Yuan, P., Li, C., Huang, Y., & Wei, W. (2014) · Nature · doi:10.1038/nature13166 · Cited by 733 · see citing works

Unravelling the structural and mechanistic basis of CRISPR–Cas systems

John van der, O. O. S. T., Westra, E. R., Jackson, R. N., & Wiedenheft, B. (2014) · Nature Reviews Microbiology · doi:10.1038/nrmicro3279 · Cited by 715 · see citing works

A split-Cas9 architecture for inducible genome editing and transcription modulation

Zetsche, B., Volz, S. E., & Zhang, F. (2015) · Nature Biotechnology · doi:10.1038/nbt.3149 · Cited by 704 · see citing works

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

Schumann, K., Lin, S., Boyer, E., Simeonov, D. R., Subramaniam, M., Gate, R. E., Haliburton, G. E., Ye, C. J., Bluestone, J. A., Doudna, J. A., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1512503112 · Cited by 702 · see citing works

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696 · see citing works

Efficient genome engineering in human pluripotent stem cells using Cas9 from <i>Neisseria meningitidis</i>

Hou, Z., Zhang, Y., Propson, N. E., Howden, S. E., Chu, L.-F., Sontheimer, E. J., & Thomson, J. A. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1313587110 · Cited by 681 · see citing works

Proteins and DNA elements essential for the CRISPR adaptation process in Escherichia coli

Yosef, I., Goren, M. G., & Qimron, U. (2012) · Nucleic Acids Research · doi:10.1093/nar/gks216 · Cited by 680 · see citing works

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

Ramakrishna, S., Kwaku Dad, A.-B., Beloor, J., Gopalappa, R., Lee, S.-K., & Kim, H. (2014) · Genome Research · doi:10.1101/gr.171264.113 · Cited by 655 · see citing works

Self‐assembled DNA nanoclews for the efficient delivery of CRISPR–Cas9 for genome editing

Sun, W., Ji, W., Hall, J. M., Hu, Q., Wang, C., Beisel, C. L., & Gu, Z. (2015) · Angewandte Chemie International Edition · doi:10.1002/anie.201506030 · Cited by 629 · see citing works

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

Wang, M., Zuris, J. A., Meng, F., Rees, H., Sun, S., Deng, P., Han, Y., Gao, X., Pouli, D., Wu, Q., et al. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1520244113 · Cited by 624 · see citing works

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

Cradick, T. J., Fine, E. J., Antico, C. J., & Bao, G. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt714 · Cited by 590 · see citing works

Direct cytosolic delivery of CRISPR/Cas9-ribonucleoprotein for efficient gene editing

Mout, R., Ray, M., Yesilbag Tonga, G., Lee, Y.-W., Tay, T., Sasaki, K., & Rotello, V. M. (2017) · ACS Nano · doi:10.1021/acsnano.6b07600 · Cited by 579 · see citing works

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

Fitzgerald, K. J., Frank-Kamenetsky, M. D., Shulga‐Morskaya, S., Liebow, A., Bettencourt, B. R., Sutherland, J. E., Hutabarat, R. M., Clausen, V. A., Karsten, V., Cehelsky, J. E., et al. (2013) · The Lancet · doi:10.1016/s0140-6736(13)61914-5 · Cited by 574 · see citing works

Generation of gene-modified mice via Cas9/RNA-mediated gene targeting

Shen, B., Zhang, J., Wu, H., Wang, J., Ma, K., Li, Z., Zhang, X., Zhang, P., & Huang, X. (2013) · Cell Research · doi:10.1038/cr.2013.46 · Cited by 556 · see citing works

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang, Y., Wang, L., Bell, P., McMenamin, D., He, Z., White, J., Yu, H., Xu, C., Morizono, H., Musunuru, K., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3469 · Cited by 556 · cited 2× · see citing works

Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

Heckl, D., Kowalczyk, M. S., Yudovich, D., Belizaire, R., Puram, R. V., McConkey, M. E., Thielke, A., Aster, J. C., Regev, A., & Ebert, B. L. (2014) · Nature Biotechnology · doi:10.1038/nbt.2951 · Cited by 555 · see citing works

Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs

Ding, Q., Regan, S. N., Xia, Y., Oostrom, L. A., Cowan, C. A., & Musunuru, K. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.03.006 · Cited by 502 · see citing works

A vector-free microfluidic platform for intracellular delivery

Sharei, A., Zoldan, J., Adamo, A., Sim, W. Y., Cho, N., Jackson, E., Mao, S., Schneider, S., Han, M.-J., Lytton‐Jean, A. K. R., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1218705110 · Cited by 497 · see citing works

Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9

Mandal, P. K., Ferreira, L. M. R., Collins, R., Meissner, T. B., Boutwell, C. L., Friesen, M., Vrbanac, V., Garrison, B. S., Stortchevoi, A., Bryder, D., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.10.004 · Cited by 475 · see citing works

Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing

Veres, A., Gosis, B. S., Ding, Q., Collins, R., Ragavendran, A., Brand, H., Erdin, S., Cowan, C. A., Talkowski, M. E., & Musunuru, K. (2014) · Cell Stem Cell · doi:10.1016/j.stem.2014.04.020 · Cited by 474 · see citing works

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

Ylä‐Herttuala, S. (2012) · Molecular Therapy · doi:10.1038/mt.2012.194 · Cited by 465 · see citing works

Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors

Wang, X., Wang, Y., Wu, X., Wang, J., Wang, Y., Qiu, Z., Chang, T., Huang, H., Lin, R.-J., & Yee, J.-K. (2015) · Nature Biotechnology · doi:10.1038/nbt.3127 · Cited by 442 · see citing works

CRISPR gene-editing tested in a person for the first time

Cyranoski, D. (2016) · Nature · doi:10.1038/nature.2016.20988 · Cited by 442 · see citing works

Generation of mutant mice by pronuclear injection of circular plasmid expressing Cas9 and single guided RNA

Mashiko, D., Fujihara, Y., Satouh, Y., Miyata, H., Isotani, A., & Ikawa, M. (2013) · Scientific Reports · doi:10.1038/srep03355 · Cited by 426 · see citing works

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

Sonntag, F., Schmidt, K., & Kleinschmidt, J. A. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1001673107 · Cited by 392 · see citing works

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector

Kabadi, A. M., Ousterout, D. G., Hilton, I. B., & Gersbach, C. A. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku749 · Cited by 371 · see citing works

Hydrodynamic Gene Delivery: Its Principles and Applications

Suda, T., & Liu, D. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300314 · Cited by 349 · see citing works

From Virus Evolution to Vector Revolution: Use of Naturally Occurring Serotypes of Adeno-associated Virus (AAV) as Novel Vectors for Human Gene Therapy

Grimm, D., & Kay, M. A. (2003) · Current Gene Therapy · doi:10.2174/1566523034578285 · Cited by 349 · see citing works

Efficient Intracellular Delivery of Native Proteins

D’Astolfo, D. S., Pagliero, R. J., Pras, A., Karthaus, W. R., Clevers, H. C., Prasad, V., Lebbink, R. J., Rehmann, H., & Geijsen, N. (2015) · Cell · doi:10.1016/j.cell.2015.03.028 · Cited by 343 · see citing works

DNA Nanoflowers for Multiplexed Cellular Imaging and Traceable Targeted Drug Delivery

Hu, R., Zhang, X., Zhao, Z., Zhu, G., Chen, T., Fu, T., & Tan, W. (2014) · Angewandte Chemie International Edition · doi:10.1002/anie.201400323 · Cited by 337 · see citing works

CRISPR–Cas systems: beyond adaptive immunity

Westra, E. R., Buckling, A., & Fineran, P. C. (2014) · Nature Reviews Microbiology · doi:10.1038/nrmicro3241 · Cited by 322 · see citing works

Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing

Kaminski, R., Chen, Y., Fischer, T., Tedaldi, E., Napoli, A., Zhang, Y., Karn, J., Hu, W., & Khalili, K. (2016) · Scientific Reports · doi:10.1038/srep22555 · Cited by 320 · see citing works

Efficient Generation of Myostatin Knock-Out Sheep Using CRISPR/Cas9 Technology and Microinjection into Zygotes

Crispo, M., Mulet, A. P., Tesson, L., Barrera, N., Cuadro, F., dos Santos-Neto, P. C., Nguyen, T. H., Crénéguy, A., Brusselle, L., Anegón, I., et al. (2015) · PLoS ONE · doi:10.1371/journal.pone.0136690 · Cited by 310 · see citing works

CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus

Ramanan, V., Shlomai, A., Cox, D. B. T., Schwartz, R. E., Michailidis, E., Bhatta, A., Scott, D. A., Zhang, F., Rice, C. M., & Bhatia, S. N. (2015) · Scientific Reports · doi:10.1038/srep10833 · Cited by 306 · see citing works

Targeting Hepatitis B Virus With CRISPR/Cas9

Seeger, C., & Sohn, J. A. (2014) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2014.68 · Cited by 294 · see citing works

Efficient CRISPR/Cas9-Mediated Genome Editing in Mice by Zygote Electroporation of Nuclease

Qin, W., Dion, S., Kutny, P. M., Zhang, Y., Cheng, A. W., Jillette, N., Malhotra, A., Geurts, A. M., Chen, Y., & Wang, H. (2015) · Genetics · doi:10.1534/genetics.115.176594 · Cited by 290 · see citing works

Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications

Friedland, A. E., Baral, R., Singhal, P., Loveluck, K., Shen, S., Sanchez, M. E., Marco, E., Gotta, G. M., Maeder, M. L., Kennedy, E. M., et al. (2015) · Genome biology · doi:10.1186/s13059-015-0817-8 · Cited by 280 · see citing works

The CRISPR/Cas Genome-Editing Tool: Application in Improvement of Crops

Khatodia, S., Bhatotia, K., Passricha, N., Khurana, S. M. P., & Tuteja, N. (2016) · Frontiers in Plant Science · doi:10.3389/fpls.2016.00506 · Cited by 267 · see citing works

In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges

Mout, R., Ray, M., Lee, Y.-W., Scaletti, F., & Rotello, V. M. (2017) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.7b00057 · Cited by 247 · see citing works

Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication

Dong, C., Qu, L., Wang, H., Wei, L., Dong, Y., & Xiong, S. (2015) · Antiviral Research · doi:10.1016/j.antiviral.2015.03.015 · Cited by 245 · see citing works

Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus

Zhen, S. M., Hua, L., Liu, Y.-H., Gao, L.-C., Fu, J., Wan, D.-Y., Dong, L.-H., Song, H.-F., & Gao, X. (2015) · Gene Therapy · doi:10.1038/gt.2015.2 · Cited by 233 · see citing works

CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

Han, X., Liu, Z., Jo, M. C., Zhang, K., Li, Y., Zeng, Z., Li, N., Zu, Y., & Qin, L. (2015) · Science Advances · doi:10.1126/sciadv.1500454 · Cited by 232 · see citing works

RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection

Wang, J., & Quake, S. R. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1410785111 · Cited by 220 · see citing works

Nonviral Genome Editing Based on a Polymer-Derivatized CRISPR Nanocomplex for Targeting Bacterial Pathogens and Antibiotic Resistance

Kang, Y. K., Kwon, K., Ryu, J. S., Lee, H. N., Park, C., & Chung, H. J. (2017) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.6b00676 · Cited by 210 · see citing works

Simple and Rapid In Vivo Generation of Chromosomal Rearrangements using CRISPR/Cas9 Technology

Blasco, R. B., Karaca, E., Ambrogio, C., Cheong, T.-C., Karayol, E., Minero, V. G., Voena, C., & Chiarle, R. (2014) · Cell Reports · doi:10.1016/j.celrep.2014.10.051 · Cited by 206 · see citing works

Delivery 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

Validation of microinjection methods for generating knockout mice by CRISPR/Cas-mediated genome engineering

Horii, T., Arai, Y., Yamazaki, M., Morita, S., Kimura, M., Itoh, M., Abe, Y., & Hatada, I. (2014) · Scientific Reports · doi:10.1038/srep04513 · Cited by 187 · see citing works

Silencing of end-joining repair for efficient site-specific gene insertion after TALEN/CRISPR mutagenesis in Aedes aegypti

Basu, S. P., Aryan, A., Overcash, J. M., Samuel, G. H., Anderson, M. A. E., Dahlem, T. J., Myles, K. M., & Adelman, Z. N. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1502370112 · Cited by 167 · see citing works

Identification of a Novel Family of Sequence Repeats among Prokaryotes

Jansen, R. K., van Embden, J. D. A., Gaastra, W., & Schouls, L. M. (2002) · OMICS A Journal of Integrative Biology · doi:10.1089/15362310252780816 · Cited by 163 · see citing works

Inhibition of zebrafish fin regeneration using in vivo electroporation of morpholinos against fgfr1 and msxb

Thummel, R., Bai, S., Sarras, M. P., Song, P., McDermott, J. P., Brewer, J., Perry, M., Zhang, X., Hyde, D. R., & Godwin, A. R. F. (2005) · Developmental Dynamics · doi:10.1002/dvdy.20630 · Cited by 144 · see citing works

Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells

Maggio, I., Holkers, M., Liu, J., Janssen, J. M., Chen, X., & Gonçalves, M. A. F. V. (2014) · Scientific Reports · doi:10.1038/srep05105 · Cited by 138 · see citing works

Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡

Paulk, N. K., Wursthorn, K., Wang, Z., Finegold, M. J., Kay, M. A., & Grompe, M. (2010) · Hepatology · doi:10.1002/hep.23481 · Cited by 135 · see citing works

Ethical issues of CRISPR technology and gene editing through the lens of solidarity

Mulvihill, J. J., Capps, B., Joly, Y., Lysaght, T., Zwart, H. A. E., & CHADWICK, R. F. (2017) · British Medical Bulletin · doi:10.1093/bmb/ldx002 · Cited by 125 · see citing works

Molecular mechanisms of CRISPR-mediated microbial immunity

Gasiūnas, G., Šinkūnas, T., & Šikšnys, V. (2013) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-013-1438-6 · Cited by 117 · see citing works

Development of a Self-Inactivating, Minimal Lentivirus Vector Based on Simian Immunodeficiency Virus

Schnell, T., Foley, P., Wirth, M., Münch, J., & Überla, K. (2000) · Human Gene Therapy · doi:10.1089/10430340050015905 · Cited by 117 · see citing works

Gain- and loss-of-function in chick embryos by electroporation

Nakamura, H., Katahira, T., Sato, T., 渡邉, 裕., & Funahashi, J. (2004) · Mechanisms of Development · doi:10.1016/j.mod.2004.05.013 · Cited by 117 · see citing works

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

Gaj, T., Epstein, B. E., & Schaffer, D. V. (2015) · Molecular Therapy · doi:10.1038/mt.2015.151 · Cited by 117 · see citing works

Generation of multi-gene knockout rabbits using the Cas9/gRNA system

Yan, Q., Zhang, Q., Yang, H., Zou, Q., Tang, C., Fan, N., & Lai, L. (2014) · Cell Regeneration · doi:10.1186/2045-9769-3-12 · Cited by 92 · see citing works

Cell-Penetrating Peptide-Mediated Delivery of Cas9 Protein and Guide RNA for Genome Editing

Suresh, B., Ramakrishna, S., & Kim, S. (2016) · Methods in molecular biology · doi:10.1007/978-1-4939-6518-2_7 · Cited by 89 · see citing works

Feasibility for a large scale mouse mutagenesis by injecting CRISPR/Cas plasmid into zygotes

Mashiko, D., Young, S. A. M., Muto, M., Kato, H., Nozawa, K., Ogawa, M., Noda, T., Kim, Y., Satouh, Y., Fujihara, Y., et al. (2013) · Development Growth & Differentiation · doi:10.1111/dgd.12113 · Cited by 82 · see citing works

CRISPR / C as9‐mediated gene knockout in the ascidian C iona intestinalis

Sasaki, H., Yoshida, K., Hozumi, A., & Sasakura, Y. (2014) · Development Growth & Differentiation · doi:10.1111/dgd.12149 · Cited by 81 · see citing works

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

Khorsandi, S. E., Bachellier, P., Weber, J. C., Greget, M., Jaeck, D., Zacharoulis, D., Rountas, C., Helmy, S. M., Helmy, A. S., Al-Waracky, M., et al. (2008) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7701119 · Cited by 69 · see citing works

Gene Therapy Delivery Systems for Enhancing Viral and Nonviral Vectors for Cardiac Diseases: Current Concepts and Future Applications

Katz, M. G., Fargnoli, A. S., Williams, R. D., & Bridges, C. R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.2517 · Cited by 67 · see citing works

Exploiting CRISPR / C as systems for biotechnology

Sampson, T. R., & Weiss, D. S. (2013) · BioEssays · doi:10.1002/bies.201300135 · Cited by 62 · see citing works

Production of knockout mice by DNA microinjection of various CRISPR/Cas9 vectors into freeze-thawed fertilized oocytes

Nakagawa, Y., Sakuma, T., Sakamoto, T., Ohmuraya, M., Nakagata, N., & Yamamoto, T. (2015) · BMC Biotechnology · doi:10.1186/s12896-015-0144-x · Cited by 55 · see citing works

Generation of Zebrafish Models by CRISPR/Cas9 Genome Editing

Hruscha, A., & Schmid, B. (2014) · Methods in molecular biology · doi:10.1007/978-1-4939-2152-2_24 · Cited by 46 · see citing works

Hydrodynamic Delivery

Al-Dosari, M. S., Knapp, J. E., & Liu, D. (2005) · Advances in genetics · doi:10.1016/s0065-2660(05)54004-5 · Cited by 44 · see citing works

3′ UTR-Dependent, miR-92-Mediated Restriction of Tis21 Expression Maintains Asymmetric Neural Stem Cell Division to Ensure Proper Neocortex Size

Fei, J., Haffner, C., & Huttner, W. Β. (2014) · Cell Reports · doi:10.1016/j.celrep.2014.03.033 · Cited by 42 · see citing works

CRISPR-Cas9 as a Powerful Tool for Efficient Creation of Oncolytic Viruses

Yuan, M., Webb, E., Lemoine, N. R., & Wang, Y. (2016) · Viruses · doi:10.3390/v8030072 · Cited by 41 · see citing works

Tissue- and time-directed electroporation of CAS9 protein–gRNA complexes in vivo yields efficient multigene knockout for studying gene function in regeneration

Fei, J., Knapp, D., Schuez, M., Murawala, P., Zou, Y., Singh, S. P., Drechsel, D. N., & Tanaka, E. M. (2016) · npj Regenerative Medicine · doi:10.1038/npjregenmed.2016.2 · Cited by 37 · see citing works

Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR/Cas9 gene editing

Khalili, K., Kaminski, R., Chen, Y., et al. (2015) · Journal of Virus Eradication · doi:10.1016/s2055-6640(20)31337-6 · Cited by 36 · see citing works

Hydrodynamic Delivery

Suda, T., & Liu, D. (2014) · Advances in genetics · doi:10.1016/bs.adgen.2014.10.002 · Cited by 22 · see citing works

Germline editing dominates DNA summit

Travis, J. P. (2015) · Science · doi:10.1126/science.350.6266.1299 · Cited by 21 · see citing works

Inhibition of zebrafish fin regeneration using in vivo electroporation of morpholinos against fgfr1 and msxb

Thummel, R., Bai, S., Sarras, M. P., et al. (2006) · Developmental Biology · doi:10.1016/j.ydbio.2006.04.095 · Cited by 16 · see citing works

324. Multiplex CRISPR/Cas9-Based Genome Engineering from a Single Lentiviral Vector

(2014) · Molecular Therapy · doi:10.1016/s1525-0016(16)35337-0 · Cited by 16 · see citing works

Mechanical Deformation of Cultured Cells with Hydrogels

Worthen, C., Rittié, L., & Fisher, G. J. (2017) · Methods in molecular biology · doi:10.1007/978-1-4939-7113-8_17 · Cited by 11 · see citing works

CGMP-compliant, clinical scale, non-viral platform for efficient gene editing using CRISPR/Cas9

Li, L., Natarajan, P., Allen, C., et al. (2014) · Cytotherapy · doi:10.1016/j.jcyt.2014.01.125 · Cited by 10 · see citing works

High-Throughput Screening and CRISPR-Cas9 Modeling of Causal Lipid-Associated Expression Quantitative Trait Locus VariantsPreprint

Raghavan, A., Wang, X., Rogov, P., Wang, L., Zhang, X., Mikkelsen, T. S., & Musunuru, K. (2016) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/056820 · Cited by 7 · see citing works

Lipid nanoparticles enabling gene therapies: from concepts to clinical utility

Kulkarni, J. A., Cullis, P. R., & van der Meel, R. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0721 · Cited by 592 · see citing works

Non-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

Strategies for nonviral nanoparticle‐based delivery of CRISPR/Cas9 therapeutics

Chen, F., Alphonse, M., & Liu, Q. (2020) · WIREs Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1609 · Cited by 197 · see citing works

RNA delivery biomaterials for the treatment of genetic and rare diseases

Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82 · see citing works

Engineering Nano‐Therapeutics to Boost Adoptive Cell Therapy for Cancer Treatment

Zheng, C., Zhang, J., Chan, H. F., Hu, H., Lv, S., Na, N., Tao, Y., & Li, M. (2021) · Small Methods · doi:10.1002/smtd.202001191 · Cited by 61 · see citing works

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues

AL Qtaish, N., Gallego, I., Villate-Beitia, I., Sainz-Ramos, M., López-Méndez, T. B., Grijalvo, S., Eritja, R., Soto-Sánchez, C., Martínez-Navarrete, G., Fernández, E., et al. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12030198 · Cited by 57 · see citing works

Novel methods in adrenal research: a metabolomics approach

Papathomas, T. G., Sun, N., Chortis, V., Taylor, A. E., Arlt, W., Richter, S., Eisenhofer, G., Ruiz-Babot, G., Guasti, L., & Walch, A. K. (2019) · Histochemistry and Cell Biology · doi:10.1007/s00418-019-01772-w · Cited by 14 · see citing works

Evolution of prime editing systems: move forward to the treatment of hereditary diseases

Volodina, O. V., Fabrichnikova, A. R., Anuchina, A. A., Mishina, O. S., Lavrov, A. V., & Smirnikhina, S. A. (2025) · Current Gene Therapy · doi:10.2174/0115665232295117240405070809 · Cited by 1 · see citing works

Elevated expression of exogenous RAD51 enhances the CRISPR/Cas9-mediated genome editing efficiency

Park, S. J., Yoon, S., Choi, E.-H., Hyeon, H., Lee, K., & Kim, K. P. (2023) · BMB Reports · doi:10.5483/bmbrep.2022-0149 · Cited by 12 · see citing works

Temperature effect on CRISPR-Cas9 mediated genome editing

Xiang, G., Zhang, X., An, C., Cheng, C., & Wang, H. (2017) · Journal of genetics and genomics/Journal of Genetics and Genomics · doi:10.1016/j.jgg.2017.03.004 · Cited by 95 · see citing works

CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases

Antony, J. S., Haque, A. K. M. A., Lamsfus‐Calle, A., Daniel-Moreno, A., Mezger, M., & Kormann, M. (2018) · Advances in Cell and Gene Therapy · doi:10.1002/acg2.10 · Cited by 18 · see citing works

Therapeutic applications of CRISPR RNA-guided genome editing

Koo, T., & Kim, J. (2016) · Briefings in Functional Genomics · doi:10.1093/bfgp/elw032 · Cited by 24 · see citing works

Efficient CRISPR/Cas9 genome editing with low off-target effects in zebrafish

Hruscha, A., Krawitz, P. M., Rechenberg, A., Heinrich, V., Hecht, J., Haass, C., & Schmid, B. (2013) · Development · doi:10.1242/dev.099085 · Cited by 466 · see citing works