CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery

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

Nanocarriers as an emerging platform for cancer therapy

Peer, D., Karp, J. M., Hong, S., Farokhzad, O. C., Margalit, R., & Langer, R. (2007) · Nature Nanotechnology · doi:10.1038/nnano.2007.387 · Cited by 8,710 · 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

A Toll-like receptor recognizes bacterial DNA

Hemmi, H., Takeuchi, O., Kawai, T., Kaisho, T., Sato, S., Sanjo, H., Matsumoto, M., Hoshino, K., Wagner, H., Takeda, K., et al. (2000) · Nature · doi:10.1038/35047123 · Cited by 6,491 · see citing works

CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., Romero, D. A., & Horvath, P. (2007) · Science · doi:10.1126/science.1138140 · Cited by 6,304 · 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

Repurposing 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

Liposomal drug delivery systems: From concept to clinical applications

Allen, T. M., & Cullis, P. R. (2013) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2012.09.037 · Cited by 4,839 · see citing works

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

Zetsche, B., Gootenberg, J. S., Abudayyeh, O. O., Slaymaker, I. M., Makarova, K. S., Essletzbichler, P., Volz, S. E., Joung, J., John van der, O. O. S. T., Regev, A., et al. (2015) · Cell · doi:10.1016/j.cell.2015.09.038 · Cited by 4,750 · 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

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

Poly(ethylene glycol) in Drug Delivery: Pros and Cons as Well as Potential Alternatives

Knop, K., Hoogenboom, R., Fischer, D., & Schubert, U. S. (2010) · Angewandte Chemie International Edition · doi:10.1002/anie.200902672 · Cited by 3,533 · see citing works

One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering

Wang, H., Yang, H., Shivalila, C. S., Dawlaty, M. M., Cheng, A. W., Zhang, F., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.04.025 · Cited by 3,495 · see citing works

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299 · 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

CRISPR-Cas systems for editing, regulating and targeting genomes

Sander, J. D., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2842 · Cited by 3,153 · see citing works

Non-viral vectors for gene-based therapy

Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123 · see citing works

Efficient genome editing in zebrafish using a CRISPR-Cas system

Hwang, W. Y., Fu, Y., Reyon, D., Maeder, M. L., Tsai, S. Q., Sander, J. D., Peterson, R. T., Yeh, J.-R. J., & Joung, J. K. (2013) · Nature Biotechnology · doi:10.1038/nbt.2501 · Cited by 2,937 · see citing works

Chitosan—A versatile semi-synthetic polymer in biomedical applications

Dash, M., Chiellini, E., Ottenbrite, R. M., & Chiellini, E. (2011) · Progress in Polymer Science · doi:10.1016/j.progpolymsci.2011.02.001 · Cited by 2,920 · 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

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

Konermann, S., Brigham, M. D., Trevino, A. E., Joung, J., Abudayyeh, O. O., Barcena, C., Hsu, P. D., Habib, N., Gootenberg, J. S., Nishimasu, H., et al. (2015) · Nature · doi:10.1038/nature14136 · Cited by 2,825 · see citing works

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

Boch, J., Scholze, H., Schornack, S., Landgraf, A., Hahn, S., Kay, S., Lahaye, T., Nickstadt, A., & Bonas, U. (2009) · Science · doi:10.1126/science.1178811 · Cited by 2,707 · see citing works

Gold nanoparticles in delivery applications☆

GHOSH, P., HAN, G., De, M., KIM, C., & Rotello, V. M. (2008) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.03.016 · Cited by 2,700 · 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

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects

Kleinstiver, B. P., Pattanayak, V., Prew, M. S., Tsai, S. Q., Nguyen, N. T., Zheng, Z., & Joung, J. K. (2016) · Nature · doi:10.1038/nature16526 · Cited by 2,673 · 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

Design and development of polymers for gene delivery

Pack, D. W., Hoffman, A. S., Pun, S., & Stayton, P. S. (2005) · Nature Reviews Drug Discovery · doi:10.1038/nrd1775 · Cited by 2,568 · 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

Accumulation of sub-100 nm polymeric micelles in poorly permeable tumours depends on size

Cabral, H., Matsumoto, Y., Mizuno, K., Chen, Q., Murakami, M., Kimura, M., Terada, Y., Kano, M. R., Miyazono, K., Uesaka, M., et al. (2011) · Nature Nanotechnology · doi:10.1038/nnano.2011.166 · Cited by 2,429 · 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

Rationally engineered Cas9 nucleases with improved specificity

Slaymaker, I. M., Gao, L., Zetsche, B., Scott, D. A., Yan, W. X., & Zhang, F. (2016) · Science · doi:10.1126/science.aad5227 · Cited by 2,414 · see citing works

Nonviral vectors for gene delivery

Mintzer, M. A., & Simanek, E. E. (2009) · Chemical Reviews · doi:10.1021/cr800409e · Cited by 2,366 · see citing works

Cas-OFFinder: A fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

Bae, S., Park, J., & Kim, J.-S. (2014) · Bioinformatics · doi:10.1093/bioinformatics/btu048 · Cited by 2,339 · see citing works

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

Abudayyeh, O. O., Gootenberg, J. S., Konermann, S., Joung, J., Slaymaker, I. M., Cox, D. B. T., Shmakov, S., Makarova, K. S., Semenova, E., Станиславович, М. Л., et al. (2016) · Science · doi:10.1126/science.aaf5573 · Cited by 2,328 · see citing works

Genome editing with engineered zinc finger nucleases

Urnov, F. D., Rebar, E. J., Holmes, M. C., Zhang, H. S., & Gregory, P. D. (2010) · Nature Reviews Genetics · doi:10.1038/nrg2842 · Cited by 2,285 · see citing works

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

Tsai, S. Q., Zheng, Z., Nguyen, N. T., Liebers, M., Topkar, V. V., Thapar, V., Wyvekens, N., Khayter, C., Iafrate, A. J., Le, L. P., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3117 · Cited by 2,268 · 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

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain.

Kim, Y. G., Cha, J., & Chandrasegaran, S. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.3.1156 · Cited by 2,099 · see citing works

A TALE nuclease architecture for efficient genome editing

Miller, J. C., Tan, S., Qiao, G., Barlow, K. A., Wang, J., Xia, D. F., Meng, X., Paschon, D. E., Leung, E., Hinkley, S. J., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1755 · Cited by 2,098 · see citing works

Altering the Genome by Homologous Recombination

Capecchi, M. R. (1989) · Science · doi:10.1126/science.2660260 · Cited by 2,037 · 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

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

Fu, Y., Sander, J. D., Reyon, D., Cascio, V. M., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2808 · Cited by 1,993 · see citing works

A Simple Cipher Governs DNA Recognition by TAL Effectors

Moscou, M., & Bogdanove, A. J. (2009) · Science · doi:10.1126/science.1178817 · Cited by 1,992 · see citing works

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers

Hilton, I. B., D’Ippolito, A. M., Vockley, C. M., Thakore, P. I., Crawford, G. E., Reddy, T. E., & Gersbach, C. A. (2015) · Nature Biotechnology · doi:10.1038/nbt.3199 · Cited by 1,878 · 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

Delivery materials for siRNA therapeutics

Kanasty, R., Dorkin, J. R., Vegas, A., & Anderson, D. (2013) · Nature Materials · doi:10.1038/nmat3765 · Cited by 1,800 · see citing works

Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR

Haeussler, M., Schönig, K., Eckert, H., Eschstruth, A., Mianné, J., Renaud, J.-B., Schneider-Maunoury, S., Shkumatava, A., Teboul, L., Kent, J., et al. (2016) · Genome biology · doi:10.1186/s13059-016-1012-2 · Cited by 1,756 · 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 · see citing works

CRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA

Marraffini, L. A., & Sontheimer, E. J. (2008) · Science · doi:10.1126/science.1165771 · Cited by 1,752 · see citing works

Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin

Bolotin, A., Quinquis, B., Sorokin, A., & Ehrlich, S. D. (2005) · Microbiology · doi:10.1099/mic.0.28048-0 · Cited by 1,733 · see citing works

Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation

Doench, J. G., Hartenian, E., Graham, D. B., Tothova, Z., Hegde, M., Smith, I., Sullender, M., Ebert, B. L., Xavier, R. J., & Root, D. E. (2014) · Nature Biotechnology · doi:10.1038/nbt.3026 · Cited by 1,617 · 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

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

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

Schwank, G., Koo, B.-K., Sasselli, V., Dekkers, J. F., Heo, I., Demircan, T., Sasaki, N., Boymans, S., Cuppen, E., van der Ent, C. K., et al. (2013) · Cell Stem Cell · doi:10.1016/j.stem.2013.11.002 · Cited by 1,424 · see citing works

RNAi-mediated gene silencing in non-human primates

Zimmermann, T. S., Lee, A. C. H., Akinc, A., Bramlage, B., Bumcrot, D., Fedoruk, M. N., Harborth, J., Heyes, J. A., Jeffs, L. B., John, M., et al. (2006) · Nature · doi:10.1038/nature04688 · Cited by 1,357 · see citing works

Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases

Cho, S. W., Kim, S., Kim, Y., Kweon, J., Kim, H. S., Bae, S., & Kim, J.-S. (2014) · Genome Research · doi:10.1101/gr.162339.113 · Cited by 1,356 · see citing works

CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies

Pourcel, C., Salvignol, G., & Vergnaud, G. (2005) · Microbiology · doi:10.1099/mic.0.27437-0 · Cited by 1,347 · 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

RNA-guided gene activation by CRISPR-Cas9-based transcription factors

Perez-Pinera, P., Kocak, D. D., Vockley, C. M., Adler, A. F., Kabadi, A. M., Polstein, L. R., Thakore, P. I., Glass, K. A., Ousterout, D. G., Leong, K. W., et al. (2013) · Nature Methods · doi:10.1038/nmeth.2600 · Cited by 1,328 · see citing works

CRISPRscan: designing highly efficient sgRNAs for CRISPR-Cas9 targeting in vivo

Moreno-Mateos, M. A., Vejnar, C. E., Beaudoin, J.-D., Fernandez, J. P., Mis, E. K., Khokha, M. K., & Giraldez, A. J. (2015) · Nature Methods · doi:10.1038/nmeth.3543 · Cited by 1,290 · see citing works

CRISPR RNA–guided activation of endogenous human genes

Maeder, M. L., Linder, S. J., Cascio, V. M., Fu, Y., Ho, Q. H., & Joung, J. K. (2013) · Nature Methods · doi:10.1038/nmeth.2598 · Cited by 1,249 · see citing works

Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems

Shmakov, S., Abudayyeh, O. O., Makarova, K. S., Wolf, Y. I., Gootenberg, J. S., Semenova, E., Станиславович, М. Л., Joung, J., Konermann, S., Severinov, K., et al. (2015) · Molecular Cell · doi:10.1016/j.molcel.2015.10.008 · Cited by 1,221 · see citing works

DNA-free genome editing in plants with preassembled CRISPR-Cas9 ribonucleoproteins

Woo, J. W., Kim, J., Kwon, S. I., Corvalán, C., Cho, S. W., Kim, H., Kim, S.-G., Kim, S.-T., Choe, S., & Kim, J.-S. (2015) · Nature Biotechnology · doi:10.1038/nbt.3389 · Cited by 1,219 · see citing works

Advances in the development of nucleoside and nucleotide analogues for cancer and viral diseases

Jordheim, L. P., Durantel, D., Zoulim, F., & Dumontet, C. (2013) · Nature Reviews Drug Discovery · doi:10.1038/nrd4010 · Cited by 1,217 · see citing works

CHOPCHOP: a CRISPR/Cas9 and TALEN web tool for genome editing

Montague, T. G., Cruz, J. M. B. S., Gagnon, J. A., Church, G. M., & Valen, E. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku410 · Cited by 1,203 · see citing works

In 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

Modeling colorectal cancer using CRISPR-Cas9–mediated engineering of human intestinal organoids

Matano, M., Date, S., Shimokawa, M., Takano, A., Fujii, M., Ohta, Y., Watanabe, T., Kanai, T., & Sato, T. (2015) · Nature Medicine · doi:10.1038/nm.3802 · Cited by 1,135 · see citing works

Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system

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

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

Jackson, A. L., & Linsley, P. S. (2009) · Nature Reviews Drug Discovery · doi:10.1038/nrd3010 · Cited by 1,092 · see citing works

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

Hendel, A., Bak, R. O., Clark, J. T., Kennedy, A. B., Ryan, D. E., Roy, S., Steinfeld, I., Lunstad, B. D., Kaiser, R. J., Wilkens, A. B., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3290 · Cited by 1,090 · see citing works

CCTop: An intuitive, flexible and reliable CRISPR/Cas9 target prediction tool

Stemmer, M., Thumberger, T., del Sol Keyer, M., Wittbrodt, J., & Mateo, J. L. (2015) · PLoS ONE · doi:10.1371/journal.pone.0124633 · Cited by 1,088 · 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

An improved zinc-finger nuclease architecture for highly specific genome editing

Miller, J. C., Holmes, M. C., Wang, J., Guschin, D. Y., Lee, Y.-L., Rupniewski, I., Beausejour, C. M., Waite, A. J., Wang, N. S., Kim, K. A., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1319 · Cited by 1,051 · see citing works

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

Tabebordbar, M., Zhu, K., Cheng, J. K. W., Chew, W. L., Widrick, J. J., Yan, W. X., Maesner, C., Wu, E. Y., Xiao, R., Ran, F. A., et al. (2016) · Science · doi:10.1126/science.aad5177 · Cited by 1,046 · 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

Therapeutic targeting of microRNAs: current status and future challenges

Li, Z., & Rana, T. M. (2014) · Nature Reviews Drug Discovery · doi:10.1038/nrd4359 · Cited by 1,028 · 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

High efficiency transformation by direct microinjection of DNA into cultured mammalian cells

Capecchi, M. R. (1980) · Cell · doi:10.1016/0092-8674(80)90358-x · Cited by 1,000 · see citing works

Digenome-seq: Genome-wide profiling of CRISPR-cas9 off-target effects in human cells

Kim, D., Bae, S., Park, J., Kim, E., Kim, S., Yu, H. R., Hwang, J., Kim, J.-I., & Kim, J.-S. (2015) · Nature Methods · doi:10.1038/nmeth.3284 · Cited by 992 · see citing works

Minimal "Self" Peptides That Inhibit Phagocytic Clearance and Enhance Delivery of Nanoparticles

Rodriguez, P. L., Harada, T., Christian, D. A., Pantano, D. A., Tsai, R. K., & Discher, D. E. (2013) · Science · doi:10.1126/science.1229568 · Cited by 986 · see citing works

TAL effectors: customizable proteins for DNA targeting

Bogdanove, A. J., & Voytas, D. F. (2011) · Science · doi:10.1126/science.1204094 · Cited by 984 · see citing works

The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic

Davis, M. E. (2009) · Molecular Pharmaceutics · doi:10.1021/mp900015y · Cited by 979 · see citing works

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

Tsai, S. Q., Wyvekens, N., Khayter, C., Foden, J. A., Thapar, V., Reyon, D., Goodwin, M. J., Aryee, M. J., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2908 · Cited by 952 · 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

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

Paquet, D., Kwart, D., Chen, A., Sproul, A., Jacob, S., Teo, S., Olsen, K. M., Gregg, A., Noggle, S., & Tessier-Lavigne, M. (2016) · Nature · doi:10.1038/nature17664 · Cited by 951 · see citing works

Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

Wu, X., Scott, D. A., Kriz, A. J., Chiu, A. C., Hsu, P. D., Dadon, D. B., Cheng, A. W., Trevino, A. E., Konermann, S., Chen, S., et al. (2014) · Nature Biotechnology · doi:10.1038/nbt.2889 · Cited by 944 · see citing works

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

Long, C., Amoasii, L., Mireault, A. A., McAnally, J., Li, H. Z., Sanchez‐Ortiz, E., Bhattacharyya, S., Shelton, J. M., Bassel‐Duby, R., & Olson, E. N. (2015) · Science · doi:10.1126/science.aad5725 · Cited by 941 · see citing works

Engineering complex synthetic transcriptional programs with CRISPR RNA scaffolds

Zalatan, J. G., Lee, M. E., Almeida, R., Gilbert, L. A., Whitehead, E. H., La Russa, M., Tsai, J. C., Weissman, J. S., Dueber, J. E., Qi, L. S., et al. (2015) · Cell · doi:10.1016/j.cell.2014.11.052 · Cited by 938 · see citing works

Highly Specific and Efficient CRISPR/Cas9-Catalyzed Homology-Directed Repair in Drosophila

Gratz, S. J., Ukken, F. P., Rubinstein, C. D., Thiede, G. H., Donohue, L. K., Cummings, A. M., & O’Connor-Giles, K. M. (2014) · Genetics · doi:10.1534/genetics.113.160713 · Cited by 900 · 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

E-CRISP: fast CRISPR target site identification

Heigwer, F., Kerr, G., & Boutros, M. (2014) · Nature Methods · doi:10.1038/nmeth.2812 · Cited by 866 · see citing works

Targeted Chromosomal Cleavage and Mutagenesis in Drosophila Using Zinc-Finger Nucleases

Bibikova, M., Golic, M., Golic, K. G., & Carroll, D. (2002) · Genetics · doi:10.1093/genetics/161.3.1169 · Cited by 860 · see citing works

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

Guilinger, J. P., Thompson, D. B., & Liu, D. R. (2014) · Nature Biotechnology · doi:10.1038/nbt.2909 · Cited by 836 · 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

Enhancing Gene Targeting with Designed Zinc Finger Nucleases

Bibikova, M. V., Beumer, K. J., Trautman, J. K., & Carroll, D. (2003) · Science · doi:10.1126/science.1079512 · Cited by 824 · see citing works

Inkjet printing as a deposition and patterning tool for polymers and inorganic particles

Tekin, E., Smith, P. J., & Schubert, U. S. (2008) · Soft Matter · doi:10.1039/b711984d · Cited by 807 · 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

Orthogonal 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

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

Kauffman, K. J., Dorkin, J. R., Yang, J. H., Heartlein, M. W., DeRosa, F., Mir, F. F., Fenton, O. S., & Anderson, D. G. (2015) · Nano Letters · doi:10.1021/acs.nanolett.5b02497 · Cited by 785 · see citing works

Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease

Kuscu, C., Arslan, S., Singh, R., Thorpe, J., & Adli, M. (2014) · Nature Biotechnology · doi:10.1038/nbt.2916 · Cited by 777 · 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 · see citing works

Engineering adeno-associated viruses for clinical gene therapy

Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761 · see citing works

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

Cheng, A. W., Wang, H., Yang, H., Shi, L., Yarden Katz, A., Theunissen, T. W., Rangarajan, S., Shivalila, C., Dadon, D. B., & Jaenisch, R. (2013) · Cell Research · doi:10.1038/cr.2013.122 · Cited by 743 · 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

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

Kormann, M. S. D., Hasenpusch, G., Aneja, M. K., Nica, G., Flemmer, A. W., Herber-Jonat, S., Huppmann, M., Mays, L. E., Illenyi, M., Schams, A., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1733 · Cited by 733 · see citing works

Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA

Long, C., McAnally, J. R., Shelton, J. M., Mireault, A. A., Bassel-Duby, R., & Olson, E. N. (2014) · Science · doi:10.1126/science.1254445 · Cited by 720 · see citing works

Microfluidic technologies for accelerating the clinical translation of nanoparticles

Valencia, P. M., Farokhzad, O. C., Karnik, R., & Langer, R. (2012) · Nature Nanotechnology · doi:10.1038/nnano.2012.168 · Cited by 704 · 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

Different behavior of branched and linear polyethylenimine for gene deliveryin vitro andin vivo

Wightman, L., Kircheis, R., R�ssler, V., Carotta, S., Ruzicka, R., Kursa, M., & Wagner, E. (2001) · The Journal of Gene Medicine · doi:10.1002/jgm.187 · Cited by 696 · see citing works

Nonviral delivery of self-amplifying RNA vaccines

Geall, A. J., Verma, A., Otten, G. R., Shaw, C. A., Hekele, A., Banerjee, K., Cu, Y., Beard, C. W., Brito, L. A., Krucker, T., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1209367109 · Cited by 675 · see citing works

Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases

Frock, R. L., Hu, J., Meyers, R. M., Ho, Y.-J., Kii, E., & Alt, F. W. (2015) · Nature Biotechnology · doi:10.1038/nbt.3101 · Cited by 673 · see citing works

State-of-the-art gene-based therapies: the road ahead

Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 672 · 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

High Efficiency, Homology-Directed Genome Editing in Caenorhabditis elegans Using CRISPR-Cas9 Ribonucleoprotein Complexes

Paix, A., Folkmann, A. W., Rasoloson, D., & Seydoux, G. (2015) · Genetics · doi:10.1534/genetics.115.179382 · Cited by 648 · see citing works

FDA-approved poly(ethylene glycol)–protein conjugate drugs

Alconcel, S. N. S., Baas, A. S., & Maynard, H. D. (2011) · Polymer Chemistry · doi:10.1039/c1py00034a · Cited by 642 · see citing works

CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences

Lin, Y., Cradick, T. J., Brown, M. T., Deshmukh, H., Ranjan, P., Sarode, N., Wile, B. M., Vertino, P. M., Stewart, F. J., & Bao, G. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku402 · Cited by 634 · see citing works

Programmable RNA recognition and cleavage by CRISPR/Cas9

O’Connell, M. R., Oakes, B. L., Sternberg, S. H., East-Seletsky, A., Kaplan, M., & Doudna, J. A. (2014) · Nature · doi:10.1038/nature13769 · Cited by 631 · see citing works

Conformational control of DNA target cleavage by CRISPR–Cas9

Sternberg, S. H., LaFrance, B., Kaplan, M., & Doudna, J. A. (2015) · Nature · doi:10.1038/nature15544 · Cited by 627 · see citing works

Sequence determinants of improved CRISPR sgRNA design

Xu, H., Xiao, T., Chen, C.-H., Li, W., Meyer, C. A., Wu, Q., Wu, D., Cong, L., Zhang, F., Liu, J. S., et al. (2015) · Genome Research · doi:10.1101/gr.191452.115 · Cited by 625 · 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

Targeted transfection by femtosecond laser

Tirlapur, U. K., & König, K. (2002) · Nature · doi:10.1038/418290a · Cited by 622 · see citing works

A 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

Correction of a genetic disease in mouse via use of CRISPR-Cas9

Wu, Y., Liang, D., Wang, Y., Bai, M., Tang, W., Bao, S., Yan, Z., Li, D., & Li, J. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.10.016 · Cited by 614 · see citing works

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

Kleinstiver, B. P., Prew, M. S., Tsai, S. Q., Nguyen, N. T., Topkar, V. V., Zheng, Z., & Joung, J. K. (2015) · Nature Biotechnology · doi:10.1038/nbt.3404 · Cited by 611 · see citing works

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

Deleavey, G. F., & Damha, M. J. (2012) · Chemistry & Biology · doi:10.1016/j.chembiol.2012.07.011 · Cited by 590 · 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

Strategies for extended serum half-life of protein therapeutics

Kontermann, R. E. (2011) · Current Opinion in Biotechnology · doi:10.1016/j.copbio.2011.06.012 · Cited by 576 · see citing works

Polymers for gene delivery across length scales

Putnam, D. A. (2006) · Nature Materials · doi:10.1038/nmat1645 · Cited by 573 · see citing works

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

Ebina, H., Misawa, N., Kanemura, Y., & Koyanagi, Y. (2013) · Scientific Reports · doi:10.1038/srep02510 · Cited by 565 · 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

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

Li, T., Huang, S., Jiang, W. Z., Wright, D. A., Spalding, M. H., Weeks, D. P., & Yang, B. (2010) · Nucleic Acids Research · doi:10.1093/nar/gkq704 · Cited by 547 · see citing works

Enhancement of transfection by physical concentration of DNA at the cell surface

Luo, D., & Saltzman, W. M. (2000) · Nature Biotechnology · doi:10.1038/78523 · Cited by 544 · see citing works

Tailoring nanocarriers for intracellular protein delivery

Gu, Z., Biswas, A., Zhao, M., & Tang, Y. (2011) · Chemical Society Reviews · doi:10.1039/c0cs00227e · Cited by 543 · see citing works

Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

Miller, J. B., Zhang, S., Kos, P., Xiong, H., Zhou, K., Perelman, S. S., Zhu, H., & Siegwart, D. J. (2017) · Angewandte Chemie International Edition · doi:10.1002/anie.201610209 · Cited by 540 · see citing works

RNA Interference in Mammalian Cells by Chemically-Modified RNA

Braasch, D. A., Jensen, S., Liu, Y., Kaur, K. J., Arar, K., White, M. A., & Corey, D. R. (2003) · Biochemistry · doi:10.1021/bi0343774 · Cited by 531 · see citing works

Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery

Schaffer, D. V., Fidelman, N. A., Dan, N. R., & Lauffenburger, D. A. (2000) · Biotechnology and Bioengineering · doi:10.1002/(sici)1097-0290(20000305)67:5 · Cited by 525 · 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

Sequence-engineered mRNA without chemical nucleoside modifications enables an effective protein therapy in large animals

Thess, A., Grund, S., Mui, B. L., Hope, M. J., Baumhof, P., Fotin-Mleczek, M., & Schlake, T. (2015) · Molecular Therapy · doi:10.1038/mt.2015.103 · Cited by 513 · see citing works

RNA aptamers as reversible antagonists of coagulation factor IXa

Rusconi, C. P., Scardino, E., Layzer, J. M., Pitoc, G. A., Ortel, T. L., Monroe, D. M., & Sullenger, B. A. (2002) · Nature · doi:10.1038/nature00963 · Cited by 512 · see citing works

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

Tsai, S. Q., & Joung, J. K. (2016) · Nature Reviews Genetics · doi:10.1038/nrg.2016.28 · Cited by 508 · 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

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

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

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

Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing

Crosetto, N., Mitra, A., Silva, M. J., Bienko, M., Dojer, N., Wang, Q., Karaca, E., Chiarle, R., Skrzypczak, M., Ginalski, K., et al. (2013) · Nature Methods · doi:10.1038/nmeth.2408 · Cited by 465 · see citing works

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

Thakore, P. I., Black, J. B., Hilton, I. B., & Gersbach, C. A. (2016) · Nature Methods · doi:10.1038/nmeth.3733 · Cited by 451 · see citing works

Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors

McCarty, D. M., Young, S. M., & Samulski, R. J. (2004) · Annual Review of Genetics · doi:10.1146/annurev.genet.37.110801.143717 · Cited by 450 · 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

A CRISPR/Cas system mediates bacterial innate immune evasion and virulence

Sampson, T. R., Saroj, S. D., Llewellyn, A. C., Tzeng, Y., & Weiss, D. S. (2013) · Nature · doi:10.1038/nature12048 · Cited by 441 · see citing works

Chemical modification and design of anti-miRNA oligonucleotides

Lennox, K. A., & Behlke, M. A. (2011) · Gene Therapy · doi:10.1038/gt.2011.100 · Cited by 432 · see citing works

Viral Vectors for Gene Therapy: Translational and Clinical Outlook.

Kotterman, M., Chalberg, T., & Schaffer, D. (2015) · Annual Review of Biomedical Engineering · doi:10.1146/annurev-bioeng-071813-104938 · Cited by 425 · see citing works

Small molecule–triggered Cas9 protein with improved genome-editing specificity

Davis, K. M., Pattanayak, V., Thompson, D. B., Zuris, J. A., & Liu, D. R. (2015) · Nature Chemical Biology · doi:10.1038/nchembio.1793 · Cited by 418 · see citing works

sgRNAcas9: A Software Package for Designing CRISPR sgRNA and Evaluating Potential Off-Target Cleavage Sites

Xie, S., Shen, B., Zhang, C., Huang, X., & Zhang, Y. (2014) · PLoS ONE · doi:10.1371/journal.pone.0100448 · Cited by 417 · see citing works

Stimuli-responsive nanomaterials for therapeutic protein delivery

Lu, Y., Sun, W., & Gu, Z. (2014) · Journal of Controlled Release · doi:10.1016/j.jconrel.2014.08.015 · Cited by 415 · see citing works

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

Cradick, T. J., Qiu, P., Lee, C. M., Fine, E. J., & Bao, G. (2014) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2014.64 · Cited by 408 · see citing works

Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach

Chari, R., Mali, P., Moosburner, M., & Church, G. M. (2015) · Nature Methods · doi:10.1038/nmeth.3473 · Cited by 394 · see citing works

The CRISPR/Cas9 system facilitates clearance of the intrahepatic HBV templates in vivo

Lin, S.-R., Yang, H.-C., Kuo, Y.-T., Liu, C.-J., Yang, T.-Y., Sung, K.-C., Lin, Y.-Y., Wang, H.-Y., Wang, C.-C., Shen, Y.-C., et al. (2014) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2014.38 · Cited by 386 · see citing works

Direct Injection of Protamine-protected mRNA: Results of a Phase 1/2 Vaccination Trial in Metastatic Melanoma Patients

Weide, B., Pascolo, S., Scheel, B., Derhovanessian, E., Pflugfelder, A., Eigentler, T. K., Pawelec, G., Hoerr, I., Rammensee, H.-G., & Garbe, C. (2009) · Journal of Immunotherapy · doi:10.1097/cji.0b013e3181a00068 · Cited by 378 · see citing works

A peptide nucleic acid–nuclear localization signal fusion that mediates nuclear transport of DNA

Brandén, L. J., Mohamed, A. J., & Smith, C. I. E. (1999) · Nature Biotechnology · doi:10.1038/11726 · Cited by 376 · see citing works

Multiplex genome engineering in human cells using all-in-one CRISPR/Cas9 vector system

Sakuma, T., Nishikawa, A., Kume, S., Chayama, K., & Yamamoto, T. (2014) · Scientific Reports · doi:10.1038/srep05400 · Cited by 376 · see citing works

WU-CRISPR: characteristics of functional guide RNAs for the CRISPR/Cas9 system

Wong, N. W., Liu, W., & Wang, X. (2015) · Genome biology · doi:10.1186/s13059-015-0784-0 · Cited by 374 · see citing works

Gene Therapy Progress and Prospects: Electroporation and other physical methods

Wells, D. J. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302337 · Cited by 373 · see citing works

Transfection efficiency of chitosan vectors: Effect of polymer molecular weight and degree of deacetylation

Huang, M., Fong, C. W., Khor, E., & Lim, L. Y. (2005) · Journal of Controlled Release · doi:10.1016/j.jconrel.2005.05.004 · Cited by 372 · see citing works

Development of an intein-mediated split-Cas9 system for gene therapy

Truong, D.-J. J., Kühner, K., Kühn, R., Werfel, S., Engelhardt, S., Wurst, W., & Ortiz, O. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv601 · Cited by 371 · see citing works

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

Bramsen, J. B., Laursen, M. B., Nielsen, A. F., Hansen, T. B., Bus, C., Langkjær, N., Babu, B. R., Højland, T., Abramov, M., Aerschot, A. A. V., et al. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp106 · Cited by 360 · see citing works

The effect of the degree of chitosan deacetylation on the efficiency of gene transfection

Kiang, T., Wen, J., Lim, H. W., & Leong, K. W. (2004) · Biomaterials · doi:10.1016/j.biomaterials.2003.12.036 · Cited by 356 · see citing works

Use of the CRISPR/Cas9 System to Produce Genetically Engineered Pigs from In Vitro-Derived Oocytes and Embryos1

Whitworth, K. M., Lee, K., Benne, J. A., Beaton, B. P., Spate, L. D., Murphy, S. L., Samuel, M., Mao, J., O’Gorman, C., Walters, E. M., et al. (2014) · Biology of Reproduction · doi:10.1095/biolreprod.114.121723 · Cited by 351 · see citing works

Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection

Li, S., Tseng, W.-C., Stolz, D. B., Wu, S.-Y., Watkins, S. C., & Huang, L. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300865 · Cited by 351 · see citing works

Recent Advances in Lentiviral Vector Development and Applications

Mátrai, J., Chuah, M., & VandenDriessche, T. (2010) · Molecular Therapy · doi:10.1038/mt.2009.319 · Cited by 349 · see citing works

Homing Endonucleases: From Microbial Genetic Invaders to Reagents for Targeted DNA Modification

Stoddard, B. L. (2011) · Structure · doi:10.1016/j.str.2010.12.003 · Cited by 346 · see citing works

Adenovirus-Mediated Somatic Genome Editing of <i>Pten</i> by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

Wang, D., Mou, H., Li, S., Li, Y., Hough, S., Tran, K., Li, J., Yin, H., Anderson, D. G., Sontheimer, E. J., et al. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.087 · Cited by 345 · 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

Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs

Smith, C., Gore, A., Yan, W., Abalde-Atristain, L., Li, Z., He, C., Wang, Y., Brodsky, R. A., Zhang, K., Cheng, L., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.06.011 · Cited by 342 · see citing works

Messenger RNA-based Vaccines With Dual Activity Induce Balanced TLR-7 Dependent Adaptive Immune Responses and Provide Antitumor Activity

Fotin-Mleczek, M., Duchardt, K. M., Lorenz, C., Pfeiffer, R., Ojkić-Zrna, S., Probst, J., & Kallen, K.-J. (2011) · Journal of Immunotherapy · doi:10.1097/cji.0b013e3181f7dbe8 · Cited by 335 · see citing works

Cas9 gRNA engineering for genome editing, activation and repression

Kiani, S., Chavez, A., Tuttle, M. L., Hall, R. N., Chari, R., Ter‐Ovanesyan, D., Qian, J., Pruitt, B. W., Beal, J., Vora, S., et al. (2015) · Nature Methods · doi:10.1038/nmeth.3580 · Cited by 335 · see citing works

Chemistry for peptide and protein PEGylation

Roberts, M. J., Bentley, M. D., & Harris, J. M. (2012) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2012.09.025 · Cited by 335 · see citing works

Cancer-Associated Protein Kinase C Mutations Reveal Kinase’s Role as Tumor Suppressor

Antal, C. E., Hudson, A., Kang, E., Zanca, C., Wirth, C., Stephenson, N. L., Trotter, E. W., Gallegos, L. L., Miller, C. J., Furnari, F. B., et al. (2015) · Cell · doi:10.1016/j.cell.2015.01.001 · Cited by 331 · see citing works

A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences

Smith, J. N. P., Grizot, S., Arnould, S., Duclert, A., Epinat, J.-C., Chames, P., Prìeto, J., Redondo, P. N., Blanco, F. J., Bravo, J., et al. (2006) · Nucleic Acids Research · doi:10.1093/nar/gkl720 · Cited by 329 · see citing works

Efficient generation of knock-in transgenic zebrafish carrying reporter/driver genes by CRISPR/Cas9-mediated genome engineering

Kimura, Y., Hisano, Y., Kawahara, A., & Higashijima, S. (2014) · Scientific Reports · doi:10.1038/srep06545 · Cited by 322 · see citing works

Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection

Ye, L., Wang, J., Beyer, A. I., Teque, F., Cradick, T. J., Qi, Z., Chang, J. C., Bao, G., Muench, M. O., Yu, J., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1407473111 · Cited by 320 · 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

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

Holkers, M., Maggio, I., Liu, J., Janssen, J. M., Miselli, F., Mussolino, C., Recchia, A., Cathomen, T., & Gonçalves, M. A. F. V. (2013) · Nucleic Acids Research · doi:10.1093/nar/gks1446 · Cited by 312 · 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

Targeted genomic rearrangements using CRISPR/Cas technology

Choi, P. S., & Meyerson, M. M. (2014) · Nature Communications · doi:10.1038/ncomms4728 · Cited by 307 · see citing works

Compacted DNA Nanoparticles Administered to the Nasal Mucosa of Cystic Fibrosis Subjects Are Safe and Demonstrate Partial to Complete Cystic Fibrosis Transmembrane Regulator Reconstitution

Konstan, M. W., Davis, P. B., Wagener, J. S., Hilliard, K. A., Stern, R. C., Milgram, L. J. H., Kowalczyk, T. H., Hyatt, S. L., Fink, T. L., Gedeon, C. R., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1255 · Cited by 298 · see citing works

Inactivation of the Human Papillomavirus E6 or E7 Gene in Cervical Carcinoma Cells by Using a Bacterial CRISPR/Cas RNA-Guided Endonuclease

Kennedy, E. M., Kornepati, A. V. R., Goldstein, M., Bogerd, H. P., Poling, B. C., Whisnant, A. W., Kastan, M. B., & Cullen, B. R. (2014) · Journal of Virology · doi:10.1128/jvi.01879-14 · Cited by 296 · see citing works

Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling

Zuckermann, M., Hovestadt, V., Knobbe-Thomsen, C. B., Zapatka, M., Northcott, P. A., Schramm, K., Belic, J., Jones, D., Tschida, B. R., Moriarity, B. S., et al. (2015) · Nature Communications · doi:10.1038/ncomms8391 · Cited by 294 · see citing works

PRINT: A Novel Platform Toward Shape and Size Specific Nanoparticle Theranostics

Perry, J. L., Herlihy, K. P., Napier, M. E., & DeSimone, J. M. (2011) · Accounts of Chemical Research · doi:10.1021/ar2000315 · Cited by 290 · see citing works

The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer

Wisse, E., Jacobs, F., Topal, B., Frederik, P. M., & Geest, B. D. (2008) · Gene Therapy · doi:10.1038/gt.2008.60 · Cited by 290 · see citing works

Increased erythropoiesis in mice injected with submicrogram quantities of pseudouridine-containing mRNA encoding erythropoietin

Karikó, K., Muramatsu, H., Keller, J. M., & Weissman, D. (2012) · Molecular Therapy · doi:10.1038/mt.2012.7 · Cited by 282 · see citing works

Asphalt, Water, and the Prebiotic Synthesis of Ribose, Ribonucleosides, and RNA

Benner, S. A., Kim, H.-J., & Carrigan, M. A. (2012) · Accounts of Chemical Research · doi:10.1021/ar200332w · Cited by 281 · 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

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

Cheng, Q., Wei, T., Farbiak, L., Johnson, L. T., Dilliard, S. A., & Siegwart, D. J. (2020) · Nature Nanotechnology · doi:10.1038/s41565-020-0669-6 · Cited by 2,383 · see citing works

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Kowalski, P. S., Rudra, A., Miao, L., & Anderson, D. G. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.012 · Cited by 1,127 · see citing works

Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR–Cas gene editing

Liu, S., Cheng, Q., Wei, T., Yu, X., Johnson, L. T., Farbiak, L., & Siegwart, D. J. (2021) · Nature Materials · doi:10.1038/s41563-020-00886-0 · Cited by 653 · see citing works

Endosomal escape and delivery of CRISPR/Cas9 genome editing machinery enabled by nanoscale zeolitic imidazolate framework

Alsaiari, S. K., Patil, S., Alyami, M., Alamoudi, K. O., Aleisa, F. A., Merzaban, J. S., Li, M., & Khashab, N. M. (2018) · Journal of the American Chemical Society · doi:10.1021/jacs.7b11754 · Cited by 517 · see citing works

mRNA-based cancer therapeutics

Liu, C., Shi, Q., Huang, X., Koo, S., Kong, N., & Tao, W. (2023) · Nature Reviews Cancer · doi:10.1038/s41568-023-00586-2 · Cited by 500 · 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

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

Liu, J., Chang, J., Jiang, Y., Meng, X., Sun, T., Mao, L., Xu, Q., & Wang, M. (2019) · Advanced Materials · doi:10.1002/adma.201902575 · Cited by 373 · see citing works

Engineering the delivery system for CRISPR-based genome editing

Glass, Z., Lee, M., Li, Y., & Xu, Q. (2018) · Trends in biotechnology · doi:10.1016/j.tibtech.2017.11.006 · Cited by 345 · see citing works

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

Wang, H.-X., Song, Z., Lao, Y.-H., Xu, X., Gong, J., Cheng, D., Chakraborty, S., Park, J. S., Li, M., Huang, D., et al. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1712963115 · Cited by 277 · see citing works

Enhancing CRISPR/Cas gene editing through modulating cellular mechanical properties for cancer therapy

Zhang, D., Wang, G., Yu, X., Wei, T., Farbiak, L., Johnson, L. T., Taylor, A. M., Xu, J., Hong, Y., Zhu, H., et al. (2022) · Nature Nanotechnology · doi:10.1038/s41565-022-01122-3 · Cited by 243 · see citing works

Methods and Applications of CRISPR-Mediated Base Editing in Eukaryotic Genomes

Hess, G. T., Tycko, J., Yao, D., & Bassik, M. C. (2017) · Molecular Cell · doi:10.1016/j.molcel.2017.09.029 · Cited by 234 · 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

Nanoscale platforms for messenger RNA delivery

Li, B., Zhang, X., & Dong, Y. (2019) · WIREs Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1530 · Cited by 170 · 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

Nanoparticles for CRISPR–Cas9 delivery

Glass, Z., Li, Y., & Xu, Q. (2017) · Nature Biomedical Engineering · doi:10.1038/s41551-017-0158-x · Cited by 69 · see citing works

Simple and Efficient Targeted Intracellular Protein Delivery with Self‐Assembled Nanovehicles for Effective Cancer Therapy

He, X., Long, Q., Zeng, Z., Yang, L., Tang, Y., & Feng, X. (2019) · Advanced Functional Materials · doi:10.1002/adfm.201906187 · Cited by 29 · 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