Delivery technologies for genome editing

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

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

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

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

Cystic Fibrosis

Webb, A. K. (2006) · Cited by 4,369

LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science · doi:10.1126/science.1088547 · Cited by 3,587

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

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

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

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

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

Cavazzana, M., Hacein‐Bey‐Abina, S., Basile, G. D. S., Gross, F., Yvon, E. S., Nusbaum, P., Selz, F., Hue, C., Dogniaux, S., Casanova, J., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,685

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

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

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

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

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

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

Cystic fibrosis

Elborn, J. S. (2016) · The Lancet · doi:10.1016/s0140-6736(16)00576-6 · Cited by 1,805

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

Playing the End Game: DNA Double-Strand Break Repair Pathway Choice

Chapman, J. R., Taylor, M. R. G., & Boulton, S. J. (2012) · Molecular Cell · doi:10.1016/j.molcel.2012.07.029 · Cited by 1,728

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

TALENs: A widely applicable technology for targeted genome editing

Joung, J. K., & Sander, J. D. (2013) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm3486 · Cited by 1,650

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

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490

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

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

Gene Editing of CCR5 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

The promises and pitfalls of RNA-interference-based therapeutics

Castanotto, D., & Rossi, J. J. (2009) · Nature · doi:10.1038/nature07758 · Cited by 1,241

A guide to genome engineering with programmable nucleases

Kim, S., & Kim, J. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3686 · Cited by 1,216

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

Chu, V. T., Weber, T., Wefers, B., Wurst, W., Sander, S., Rajewsky, K., & Kühn, R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3198 · Cited by 1,210

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

Lin, S., Staahl, B. T., Alla, R. K., & Doudna, J. A. (2014) · eLife · doi:10.7554/elife.04766 · Cited by 1,189

Gene therapy returns to centre stage

Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184

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

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

Maruyama, T., Dougan, S. K., Truttmann, M. C., Bilate, A. M., Ingram, J. R., & Ploegh, H. L. (2015) · Nature Biotechnology · doi:10.1038/nbt.3190 · Cited by 1,137

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

Liang, P., Xu, Y., Zhang, X., Ding, C., Huang, R., Zhang, Z., Lv, J., Xie, X., Chen, Y., Li, Y., et al. (2015) · Protein & Cell · doi:10.1007/s13238-015-0153-5 · Cited by 1,135

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

Regulation of Homologous Recombination in Eukaryotes

Heyer, W.-D., Ehmsen, K. T., & Liu, J. (2010) · Annual Review of Genetics · doi:10.1146/annurev-genet-051710-150955 · Cited by 1,066

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

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

Precise genome modification in the crop species Zea mays using zinc-finger nucleases

Shukla, V. K., Doyon, Y., Miller, J. C., DeKelver, R. C., Moehle, E. A., Worden, S. E., Mitchell, J. C., Arnold, N. L., Gopalan, S. M., Meng, X., et al. (2009) · Nature · doi:10.1038/nature07992 · Cited by 983

Mechanism and regulation of human non-homologous DNA end-joining

Lieber, M. R., Ma, Y., Pannicke, U., & Schwarz, K. (2003) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm1202 · Cited by 973

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 973

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

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

The future of cancer treatment: immunomodulation, CARs and combination immunotherapy

Khalil, D. N., Smith, E. L., Brentjens, R. J., & Wolchok, J. D. (2016) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2016.25 · Cited by 929

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

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845

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

Rare-disease genetics in the era of next-generation sequencing: discovery to translation

Boycott, K. M., Vanstone, M. R., Bulman, D. E., & MacKenzie, A. (2013) · Nature Reviews Genetics · doi:10.1038/nrg3555 · Cited by 804

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

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

Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease.

Rouet, P., Smih, F., & Jasin, M. (1994) · Molecular and Cellular Biology · doi:10.1128/mcb.14.12.8096 · Cited by 739

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

A prudent path forward for genomic engineering and germline gene modification

Baltimore, D., Berg, P., Botchan, M., Carroll, D., Charo, R. A., Church, G., Corn, J. E., Daley, G. Q., Doudna, J. A., Fenner, M., et al. (2015) · Science · doi:10.1126/science.aab1028 · Cited by 733

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 722

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

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

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

Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 699

Engineering targeted viral vectors for gene therapy

Waehler, R., Russell, S. J., & Curiel, D. T. (2007) · Nature Reviews Genetics · doi:10.1038/nrg2141 · Cited by 687

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

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

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

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

Genome-wide inactivation of porcine endogenous retroviruses (PERVs)

Yang, L., Güell, M., Niu, D., George, H., Lesha, E., Grishin, D., Aach, J., Shrock, E. L., Xu, W., Poci, J., et al. (2015) · Science · doi:10.1126/science.aad1191 · Cited by 611

Quality control of eukaryotic mRNA: safeguarding cells from abnormal mRNA functionPreprint

Isken, O., & Maquat, L. E. (2007) · Genes & Development · doi:10.1101/gad.1566807 · Cited by 609

Gene therapy for Wiskott-Aldrich syndrome–long-term efficacy and genotoxicity

Braun, C. J., Boztug, K., Paruzynski, A., Witzel, M., Schwarzer, A., Rothe, M., Modlich, U., Beier, R., Göhring, G., Steinemann, D., et al. (2014) · Science Translational Medicine · doi:10.1126/scitranslmed.3007280 · Cited by 562

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

Targeted genome editing in human repopulating haematopoietic stem cells

Genovese, P., Schiroli, G., Escobar, G., Di Tomaso, T., Firrito, C., Calabria, A., Moi, D., Mazzieri, R., Bonini, C., Holmes, M. C., et al. (2014) · Nature · doi:10.1038/nature13420 · Cited by 555

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., et al. (2011) · Nature · doi:10.1038/nature10177 · Cited by 548

An unbiased genome-wide analysis of zinc-finger nuclease specificity

Gabriel, R., Lombardo, A., Arens, A., Miller, J. C., Genovese, P., Kaeppel, C., Nowrouzi, A., Bartholomae, C. C., Wang, J., Friedman, G., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1948 · Cited by 526

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

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

The origins of the identification and isolation of hematopoietic stem cells, and their capability to induce donor-specific transplantation tolerance and treat autoimmune diseases

Weissman, I. L., & Shizuru, J. A. (2008) · Blood · doi:10.1182/blood-2008-08-078220 · Cited by 478

High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

Chen, F., Pruett‐Miller, S. M., Huang, Y., Gjoka, M., Duda, K., Taunton, J., Collingwood, T. N., Frödin, M., & Davis, G. D. (2011) · Nature Methods · doi:10.1038/nmeth.1653 · Cited by 475

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

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

Provasi, E., Genovese, P., Lombardo, A., Magnani, Z., Liu, P.-Q., Reik, A., Chu, V., Paschon, D. E., Zhang, L., Kuball, J., et al. (2012) · Nature Medicine · doi:10.1038/nm.2700 · Cited by 448

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

Safe harbours for the integration of new DNA in the human genome

Sadelain, M., Papapetrou, E. P., & Bushman, F. D. (2012) · Nature Reviews Cancer · doi:10.1038/nrc3179 · Cited by 441

A library of TAL effector nucleases spanning the human genome

Kim, Y., Kweon, J., Kim, A., Chon, J. K., Yoo, J. Y., Kim, H. J., Kim, S., Lee, C., Jeong, E., Chung, E., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2517 · Cited by 379

Viral vectors: from virology to transgene expression

Bouard, D., Alazard-Dany, N., & Cosset, F. (2008) · British Journal of Pharmacology · doi:10.1038/bjp.2008.349 · Cited by 377

Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases

Moehle, E. A., Rock, J. M., Lee, Y.-L., Jouvenot, Y., DeKelver, R. C., Gregory, P. D., Urnov, F. D., & Holmes, M. C. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0611478104 · Cited by 371

Mutagenesis and Oncogenesis by Chromosomal Insertion of Gene Transfer Vectors

Baum, C., Kustikova, O. S., Modlich, U., Li, Z., & Fehse, B. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.253 · Cited by 369

Enhanced Antitumor Activity of T Cells Engineered to Express T-Cell Receptors with a Second Disulfide Bond

Cohen, C. J., Li, Y. F., El‐Gamil, M., Robbins, P. F., Rosenberg, S. A., & Morgan, R. A. (2007) · Cancer Research · doi:10.1158/0008-5472.can-06-3986 · Cited by 363

Introduction of Double-Strand Breaks into the Genome of Mouse Cells by Expression of a Rare-Cutting Endonuclease

Rouet, P., Smih, F., & Jasin, M. (1994) · Molecular and Cellular Biology · doi:10.1128/mcb.14.12.8096-8106.1994 · Cited by 363

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

Adenovirus-Mediated Somatic Genome Editing of Pten 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

Efficient Intracellular Delivery of Native Proteins

D’Astolfo, D. S., Pagliero, R. J., Pras, A., Karthaus, W. R., Clevers, H., Prasad, V., Lebbink, R. J., Rehmann, H., & Geijsen, N. (2015) · Cell · doi:10.1016/j.cell.2015.03.028 · Cited by 338

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genomePreprint

DeKelver, R. C., Choi, V. M., Moehle, E. A., Paschon, D. E., Hockemeyer, D. F., Meijsing, S. H., Sancak, Y., Cui, X., Steine, E. J., Miller, J. C., et al. (2010) · Genome Research · doi:10.1101/gr.106773.110 · Cited by 316

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

Torikai, H., Reik, A., Soldner, F., Warren, E. H., Yuen, C. H.-L., Zhou, Y., Crossland, D. L., Huls, H. M., Littman, N., Zhang, Z., et al. (2013) · Blood · doi:10.1182/blood-2013-03-478255 · Cited by 312

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins

Gaj, T., Guo, J., Kato, Y., Sirk, S. J., & Barbas, C. F. (2012) · Nature Methods · doi:10.1038/nmeth.2030 · Cited by 306

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

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