Genome-editing Technologies for Gene and Cell Therapy

Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases

Guo, J., Gaj, T., & Barbas, C. F. (2010) · Journal of Molecular Biology · doi:10.1016/j.jmb.2010.04.060 · Cited by 206

Human zinc fingers as building blocks in the construction of artificial transcription factors

Bae, K.-H., Do Kwon, Y., Shin, H.-C., Hwang, M.-S., Ryu, E.-H., Park, K.-S., Yang, H.-Y., Lee, D.-K., Lee, Y., Park, J., et al. (2003) · Nature Biotechnology · doi:10.1038/nbt796 · Cited by 202

High-resolution structures of variant Zif268–DNA complexes: implications for understanding zinc finger–DNA recognition

Elrod-Erickson, M., Benson, T. E., & Pabo, C. O. (1998) · Structure · doi:10.1016/s0969-2126(98)00047-1 · Cited by 202

Building zinc fingers by selection: toward a therapeutic application.

Wu, H., Yang, W. P., & Barbas, C. F. (1995) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.92.2.344 · Cited by 195

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

Crane, A. M., Kramer, P., Bui, J. H., Chung, W. J., Li, X. S., Gonzalez-Garay, M. L., Hawkins, F., Liao, W., Mora, D., Choi, S., et al. (2015) · Stem Cell Reports · doi:10.1016/j.stemcr.2015.02.005 · Cited by 193

A rapid, generally applicable method to engineer zinc fingers illustrated by targeting the HIV-1 promoter

Isalan, M., Klug, A., & Choo, Y. (2001) · Nature Biotechnology · doi:10.1038/90264 · Cited by 191

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

Huang, X., Wang, Y., Yan, W., Smith, C., Ye, Z., Wang, J., Gao, Y., Mendelsohn, L., & Cheng, L. (2015) · Stem Cells · doi:10.1002/stem.1969 · Cited by 190

Chimeric restriction endonuclease.

Kim, Y. G., & Chandrasegaran, S. (1994) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.91.3.883 · Cited by 189

Assembly of custom TALE-type DNA binding domains by modular cloning

Morbitzer, R., Elsaesser, J., Hausner, J., & Lahaye, T. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr151 · Cited by 185

Zinc-finger nucleases as a novel therapeutic strategy for targeting hepatitis B virus DNAs

Cradick, T. J., Keck, K., Bradshaw, S., Jamieson, A. C., & McCaffrey, A. P. (2010) · Molecular Therapy · doi:10.1038/mt.2010.20 · Cited by 184

Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases

Li, L., Krymskaya, L., Wang, J., Henley, J., Rao, A., Cao, L.-F., Tran, C.-A., Torres-Coronado, M., Gardner, A., Gonzalez, N., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.65 · Cited by 181

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

Ousterout, D. G., Perez-Pinera, P., Thakore, P. I., Kabadi, A. M., Brown, M. T., Qin, X., Fedrigo, O., Mouly, V., Tremblay, J. P., & Gersbach, C. A. (2013) · Molecular Therapy · doi:10.1038/mt.2013.111 · Cited by 181

Robust ZFN-mediated genome editing in adult hemophilic mice

Anguela, X. M., Sharma, R., Doyon, Y., Miller, J. C., Li, H., Haurigot, V., Rohde, M. E., Wong, S. Y., Davidson, R. J., Zhou, S., et al. (2013) · Blood · doi:10.1182/blood-2013-04-497354 · Cited by 178

Analysis of zinc fingers optimized via phage display: evaluating the utility of a recognition code 1 1Edited by P. E. Wright

Wolfe, S. A., Greisman, H. A., Ramm, E. I., & Pabo, C. O. (1999) · Journal of Molecular Biology · doi:10.1006/jmbi.1998.2421 · Cited by 177

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

Boissel, S., Jarjour, J., Astrakhan, A., Adey, A., Gouble, A., Duchateau, P., Shendure, J., Stoddard, B. L., Certo, M. T., Baker, D., et al. (2014) · Nucleic Acids Research · doi:10.1093/nar/gkt1224 · Cited by 173

Development of Zinc Finger Domains for Recognition of the 5′-CNN-3′ Family DNA Sequences and Their Use in the Construction of Artificial Transcription Factors

Dreier, B., Fuller, R. P., Segal, D. J., Lund, C. V., Blancafort, P., Huber, A., Koksch, B., & Barbas, C. F. (2005) · Journal of Biological Chemistry · doi:10.1074/jbc.m506654200 · Cited by 171

Validated Zinc Finger Protein Designs for All 16 GNN DNA Triplet Targets

Liu, Q., Xia, Z., & Case, C. C. (2002) · Journal of Biological Chemistry · doi:10.1074/jbc.m110669200 · Cited by 164

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

Grizot, S., Smith, J. N. P., Daboussi, F., Prìeto, J., Redondo, P. N., Merino, N., Villate, M., Thomas, S., Lemaire, L., Montoya, G., et al. (2009) · Nucleic Acids Research · doi:10.1093/nar/gkp548 · Cited by 162

Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases

Redondo, P. N., Prìeto, J., Muñoz, I. G., Alibés, A., Stricher, F., Serrano, L., Cabaniols, J., Daboussi, F., Arnould, S., Perez, C., et al. (2008) · Nature · doi:10.1038/nature07343 · Cited by 159

An efficient antiviral strategy for targeting hepatitis B virus genome using transcription activator-like effector nucleases

Chen, J., Zhang, W., Lin, J., Wang, F., Wu, M., Chen, C., Zheng, Y., Peng, X., Li, J., & Yuan, Z. (2014) · Molecular Therapy · doi:10.1038/mt.2013.212 · Cited by 159

Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks

Porteus, M. H., Cathomen, T., Weitzman, M. D., & Baltimore, D. (2003) · Molecular and Cellular Biology · doi:10.1128/mcb.23.10.3558-3565.2003 · Cited by 159

Inhibition of hepatitis B virus by the CRISPR/Cas9 system via targeting the conserved regions of the viral genome

Liu, X., Hao, R., Chen, S., Guo, D., & Chen, Y. Z. (2015) · Journal of General Virology · doi:10.1099/vir.0.000159 · Cited by 155

Engineered Viruses as Genome Editing Devices

Chen, X., & Gonçalves, M. A. F. V. (2015) · Molecular Therapy · doi:10.1038/mt.2015.164 · Cited by 154

Cell-Penetrating Peptide-Mediated Delivery of TALEN Proteins via Bioconjugation for Genome Engineering

Liu, J., Gaj, T., Patterson, J. T., Sirk, S. J., & Barbas, C. F. (2014) · PLoS ONE · doi:10.1371/journal.pone.0085755 · Cited by 151

Engineered I-CreI Derivatives Cleaving Sequences from the Human XPC Gene can Induce Highly Efficient Gene Correction in Mammalian Cells

Arnould, S., Perez, C., Cabaniols, J., Smith, J. N. P., Gouble, A., Grizot, S., Epinat, J.-C., Duclert, A., Duchateau, P. N., & Pâques, F. (2007) · Journal of Molecular Biology · doi:10.1016/j.jmb.2007.04.079 · Cited by 151

Synergy between adjacent zinc fingers in sequence-specific DNA recognition

Isalan, M., Choo, Y., & Klug, A. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.11.5617 · Cited by 149

TALEN-mediated editing of endogenous T-cell receptors facilitates efficient reprogramming of T lymphocytes by lentiviral gene transfer

Berdien, B., Mock, U., Atanackovic, D., & Fehse, B. (2014) · Gene Therapy · doi:10.1038/gt.2014.26 · Cited by 149

High-efficiency targeted editing of large viral genomes by RNA-guided nucleases

Bi, Y., Sun, L., Gao, D., Ding, C., Li, Z., Li, Y., Cun, W., & Li, Q. (2014) · PLoS Pathogens · doi:10.1371/journal.ppat.1004090 · Cited by 148

Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage

Josephs, E. A., Koçak, D. D., Fitzgibbon, C. J., McMenemy, J., Gersbach, C. A., & Marszałek, P. E. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv892 · Cited by 147

Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium

McNeer, N. A., Anandalingam, K. K., Fields, R. J., Caputo, C. R., Kopic, S., Gupta, A., Quijano, E., Polikoff, L. A., Kong, Y., Bahal, R., et al. (2015) · Nature Communications · doi:10.1038/ncomms7952 · Cited by 144

Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases

Wilen, C. B., Wang, J., Tilton, J. C., Miller, J. C., Kim, K. A., Rebar, E. J., Sherrill-Mix, S. A., Patro, S. C., Secreto, A. J., Jordan, A. P. O., et al. (2011) · PLoS Pathogens · doi:10.1371/journal.ppat.1002020 · Cited by 142

Adenoviral vector DNA for accurate genome editing with engineered nucleases

Holkers, M., Maggio, I., Henriques, S. F. D., Janssen, J. M., Cathomen, T., & Gonçalves, M. A. F. V. (2014) · Nature Methods · doi:10.1038/nmeth.3075 · Cited by 138

Review and update on the molecular basis of Leber congenital amaurosis

Chacon-Camacho, O. F., & Zenteno, J. C. (2015) · World Journal of Clinical Cases · doi:10.12998/wjcc.v3.i2.112 · Cited by 137

Synthetic Zinc Finger Proteins: The Advent of Targeted Gene Regulation and Genome Modification Technologies

Gersbach, C. A., Gaj, T., & Barbas, C. F. (2014) · Accounts of Chemical Research · doi:10.1021/ar500039w · Cited by 136

Functional footprinting of regulatory DNA

Vierstra, J., Reik, A., Chang, K.-H., Stehling-Sun, S., Zhou, Y., Hinkley, S. J., Paschon, D. E., Zhang, L., Psatha, N., Bendana, Y. R., et al. (2015) · Nature Methods · doi:10.1038/nmeth.3554 · Cited by 133

CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype

Burnight, E. R., Wiley, L. A., Drack, A. V., Braun, T. A., Anfinson, K. R., Kaalberg, E. E., Halder, J. A., Affatigato, L. M., Mullins, R. F., Stone, E. M., et al. (2014) · Gene Therapy · doi:10.1038/gt.2014.39 · Cited by 128

Human Gene Targeting by Adeno-Associated Virus Vectors Is Enhanced by DNA Double-Strand Breaks

Miller, D. G., Petek, L. M., & Russell, D. W. (2003) · Molecular and Cellular Biology · doi:10.1128/mcb.23.10.3550-3557.2003 · Cited by 128

An optimized two-finger archive for ZFN-mediated gene targeting

Gupta, A., Christensen, R. G., Rayla, A. L., Lakshmanan, A., Stormo, G. D., & Wolfe, S. A. (2012) · Nature Methods · doi:10.1038/nmeth.1994 · Cited by 125

Scanning the human genome with combinatorial transcription factor libraries

Blancafort, P., Magnenat, L., & Barbas, C. F. (2003) · Nature Biotechnology · doi:10.1038/nbt794 · Cited by 125

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 125

AAV-Mediated Delivery of Zinc Finger Nucleases Targeting Hepatitis B Virus Inhibits Active Replication

Weber, N. D., Stone, D., Sedlak, R. H., Feelixge, H. S. D. S., Roychoudhury, P., Schiffer, J. T., Aubert, M., & Jerome, K. R. (2014) · PLoS ONE · doi:10.1371/journal.pone.0097579 · Cited by 122

Mutations altering the cleavage specificity of a homing endonuclease

Seligman, L. M. (2002) · Nucleic Acids Research · doi:10.1093/nar/gkf495 · Cited by 122

Comprehensive DNA Recognition through Concerted Interactions from Adjacent Zinc Fingers

Isalan, M., Klug, A., & Choo, Y. (1998) · Biochemistry · doi:10.1021/bi981358z · Cited by 121

Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells

Asuri, P., Bartel, M. A., Vazin, T., Jang, J.-H., Wong, T. B., & Schaffer, D. V. (2012) · Molecular Therapy · doi:10.1038/mt.2011.255 · Cited by 119

Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

Ousterout, D. G., Kabadi, A. M., Thakore, P. I., Pérez‐Piñera, P., Brown, M. T., Majoros, W. H., Reddy, T. E., & Gersbach, C. A. (2014) · Molecular Therapy · doi:10.1038/mt.2014.234 · Cited by 119

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma

Beane, J. D., Lee, G., Zheng, Z., Mendel, M. C., Abate‐Daga, D., Bharathan, M., Black, M. A., Gandhi, N. H., Yu, Z., Chandran, S. S., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.71 · Cited by 114

Chromatin regulation at the frontier of synthetic biology

Keung, A. J., Joung, J. K., Khalil, A. S., & Collins, J. J. (2015) · Nature Reviews Genetics · doi:10.1038/nrg3900 · Cited by 112

Living the PCSK9 Adventure: from the Identification of a New Gene in Familial Hypercholesterolemia Towards a Potential New Class of Anticholesterol Drugs

Abifadel, M., Elbitar, S., Khoury, P. E., Ghaleb, Y., Chemaly, M., Moussalli, M.-L., Rabès, J., Varret, M., & Boileau, C. (2014) · Current Atherosclerosis Reports · doi:10.1007/s11883-014-0439-8 · Cited by 111

Rapid and highly efficient construction of TALE-based transcriptional regulators and nucleases for genome modification

Li, L., Piatek, M. J., Atef, A., Piatek, A., Wibowo, A. T., Fang, X., Sabir, J. S. M., Zhu, J., & Mahfouz, M. M. (2012) · Plant Molecular Biology · doi:10.1007/s11103-012-9875-4 · Cited by 111

Compact designer TALENs for efficient genome engineering

Beurdeley, M., Bietz, F., Li, J., Thomas, S., Stoddard, T., Juillerat, A., Zhang, F., Voytas, D. F., Duchateau, P. N., & Silva, G. H. (2013) · Nature Communications · doi:10.1038/ncomms2782 · Cited by 111

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion

Şöllü, C., Pars, K., Cornu, T. I., Thibodeau-Beganny, S., Maeder, M. L., Joung, J. K., Heilbronn, R., & Cathomen, T. (2010) · Nucleic Acids Research · doi:10.1093/nar/gkq720 · Cited by 110

Enabling functional genomics with genome engineeringPreprint

Hilton, I. B., & Gersbach, C. A. (2015) · Genome Research · doi:10.1101/gr.190124.115 · Cited by 107

Gene targeting in vivo by adeno‐associated virus vectors

Miller, D. G., Wang, P.-R., Petek, L. M., Hirata, R. K., Sands, M. S., & Russell, D. W. (2006) · Nature Biotechnology · doi:10.1038/nbt1231 · Cited by 105

Targeted chromosomal deletions and inversions in zebrafishPreprint

Gupta, A., Hall, V. L., Kok, F. O., Shin, M., McNulty, J. C., Lawson, N. D., & Wolfe, S. A. (2013) · Genome Research · doi:10.1101/gr.154070.112 · Cited by 105

Redesigning the DNA‐binding specificity of a zinc finger protein: A data base‐guided approach

Desjarlais, J. R., & Berg, J. M. (1992) · Proteins Structure Function and Bioinformatics · doi:10.1002/prot.340120202 · Cited by 103

Homing endonuclease I-CreI derivatives with novel DNA target specificities

Rosen, L. E., Morrison, H. A., Masri, S., Brown, M. J., Springstubb, B., Sussman, D. R., Stoddard, B. L., & Seligman, L. M. (2006) · Nucleic Acids Research · doi:10.1093/nar/gkl645 · Cited by 99

Highly active zinc-finger nucleases by extended modular assemblyPreprint

Bhakta, M. S., Henry, I. M., Ousterout, D. G., Das, K. T., Lockwood, S. H., Meckler, J. F., Wallen, M. C., Zykovich, A., Yu, Y., Leo, H., et al. (2012) · Genome Research · doi:10.1101/gr.143693.112 · Cited by 97

Targeted Gene Therapy of Xeroderma Pigmentosum Cells Using Meganuclease and TALEN™

Dupuy, A., Valton, J., Leduc, S., Armier, J., Galetto, R., Gouble, A., Lebuhotel, C., Stary, A., Pâques, F., Duchateau, P. N., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0078678 · Cited by 94

Expanding the scope of site‐specific recombinases for genetic and metabolic engineering

Gaj, T., Sirk, S. J., & Barbas, C. F. (2013) · Biotechnology and Bioengineering · doi:10.1002/bit.25096 · Cited by 91

Preassembled zinc-finger arrays for rapid construction of ZFNs

Kim, S., Lee, M., Kim, H., Kang, M., & Kim, J. (2010) · Nature Methods · doi:10.1038/nmeth0111-7a · Cited by 90

Modular system for the construction of zinc-finger libraries and proteins

Gonzalez, B., Schwimmer, L. J., Fuller, R. P., Ye, Y., Asawapornmongkol, L., & Barbas, C. F. (2010) · Nature Protocols · doi:10.1038/nprot.2010.34 · Cited by 89

Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells

Chang, C.-J., & Bouhassira, E. E. (2012) · Blood · doi:10.1182/blood-2012-03-420703 · Cited by 85

Huntingtin‐lowering strategies in Huntington's disease: Antisense oligonucleotides, small RNAs, and gene editing

D, N. A. M., & DiFiglia, M. (2014) · Movement Disorders · doi:10.1002/mds.26020 · Cited by 84

Efficient Gene Disruption in Cultured Primary Human Endothelial Cells by CRISPR/Cas9

Abrahimi, P., Chang, W. G., Kluger, M. S., Qyang, Y., Tellides, G., Saltzman, W. M., & Pober, J. S. (2015) · Circulation Research · doi:10.1161/circresaha.117.306290 · Cited by 82

Isolation and Characterization of New Homing Endonuclease Specificities at Individual Target Site Positions

Sussman, D. R., Chadsey, M., Fauce, S., Engel, A., Bruett, A., Monnat, R., Stoddard, B. L., & Seligman, L. M. (2004) · Journal of Molecular Biology · doi:10.1016/j.jmb.2004.07.031 · Cited by 78

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

Melo, S. P., Lisowski, L., Bashkirova, E., Zhen, H. H., Chu, K. C., Keene, D. R., Marinkovich, M. P., Kay, M. A., & Oro, A. E. (2014) · Molecular Therapy · doi:10.1038/mt.2013.290 · Cited by 76

Meganuclease-mediated Inhibition of HSV1 Infection in Cultured Cells

Grosse, S. M., Huot, N., Mahiet, C., Arnould, S., Barradeau, S., Clerre, D. L., Chion-Sotinel, I., Jacqmarcq, C., Chapellier, B., Ergani, A., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.302 · Cited by 75

Generation of an HIV resistant T-cell line by targeted “stacking” of restriction factors

Voit, R. A., McMahon, M. A., Sawyer, S. L., & Porteus, M. H. (2013) · Molecular Therapy · doi:10.1038/mt.2012.284 · Cited by 74

Targeted gene therapy and cell reprogramming in Fanconi anemia

Río, P., Baños, R., Lombardo, A., Bustamante, O. Q., Alvarez, L. A., Garate, Z., Genovese, P., Almarza, E., Valeri, A., Díez, B., et al. (2014) · EMBO Molecular Medicine · doi:10.15252/emmm.201303374 · Cited by 73

A CRISPR design for next-generation antimicrobials

Beisel, C. L., Gomaa, A. A., & Barrangou, R. (2014) · Genome biology · doi:10.1186/s13059-014-0516-x · Cited by 71

TALEN Knockout of the PSIP1 Gene in Human Cells: Analyses of HIV-1 Replication and Allosteric Integrase Inhibitor Mechanism

Fadel, H. J., Morrison, J. H., Saenz, D. T., Fuchs, J. R., Kvaratskhelia, M., Ekker, S. C., & Poeschla, E. M. (2014) · Journal of Virology · doi:10.1128/jvi.01397-14 · Cited by 70

Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In

Popplewell, L. J., Koo, T., Leclerc, X., Duclert, A., Mamchaoui, K., Gouble, A., Mouly, V., Voït, T., Pâques, F., Cédrone, F., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.081 · Cited by 69

Versatile and Efficient Genome Editing in Human Cells by Combining Zinc-Finger Nucleases With Adeno-Associated Viral Vectors

Händel, E.-M., Gellhaus, K., Khan, K., Bednarski, C., Cornu, T. I., Müller-Lerch, F., Kotin, R. M., Heilbronn, R., & Cathomen, T. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.140 · Cited by 66

Integrase-defective Lentiviral Vectors as a Delivery Platform for Targeted Modification of Adenosine Deaminase Locus

Joglekar, A. V., Hollis, R. P., Kuftinec, G., Senadheera, S., Chan, R., & Kohn, D. B. (2013) · Molecular Therapy · doi:10.1038/mt.2013.106 · Cited by 65

Bacterial CRISPR/Cas DNA endonucleases: A revolutionary technology that could dramatically impact viral research and treatment

Kennedy, E. M., & Cullen, B. R. (2015) · Virology · doi:10.1016/j.virol.2015.02.024 · Cited by 64

Monomeric site-specific nucleases for genome editing

Kleinstiver, B. P., Wolfs, J. M., Kolaczyk, T., Roberts, A. K., Hu, S. X., & Edgell, D. R. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1117984109 · Cited by 62

Correction of the Crb1 rd8 Allele and Retinal Phenotype in C57BL/6N Mice Via TALEN-Mediated Homology-Directed Repair

Low, B. E., Krebs, M. P., Joung, J. K., Tsai, S. Q., Nishina, P. M., & Wiles, M. V. (2013) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.13-13278 · Cited by 62

Gene therapy for Huntington's disease

Ramaswamy, S., & Kordower, J. H. (2011) · Neurobiology of Disease · doi:10.1016/j.nbd.2011.12.030 · Cited by 61

Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination

Coluccio, A., Miselli, F., Lombardo, A., Marconi, A., Tagliazucchi, G. M., Gonçalves, M. A. F. V., Pincelli, C., Maruggi, G., Río, M. D., Naldini, L., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.143 · Cited by 56

In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites

Sander, J. D., Ramirez, C. L., Linder, S. J., Pattanayak, V., Shoresh, N., Ku, M., Foden, J. A., Reyon, D., Bernstein, B., Liu, D. R., et al. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt716 · Cited by 55

In vitro Inactivation of Latent HSV by Targeted Mutagenesis Using an HSV-specific Homing Endonuclease

Aubert, M., Boyle, N. M., Stone, D., Stensland, L., Huang, M.-L. W., Magaret, A., Galetto, R., Rawlings, D. J., Scharenberg, A. M., & Jerome, K. R. (2014) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.75 · Cited by 50

Integration of drug, protein, and gene delivery systems with regenerative medicine

Lorden, E. R., Levinson, H., & Leong, K. W. (2013) · Drug Delivery and Translational Research · doi:10.1007/s13346-013-0165-8 · Cited by 49

An Integrated Chip for the High‐Throughput Synthesis of Transcription Activator‐like Effectors

Wang, Z., Li, J., Huang, H., Wang, G., Jiang, M., Yin, S., Sun, C., Zhang, H., Zhuang, F., & Xi, J. (2012) · Angewandte Chemie International Edition · doi:10.1002/anie.201203597 · Cited by 48

Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair

Hirsch, M. L., Green, L., Porteus, M. H., & Samulski, R. J. (2010) · Gene Therapy · doi:10.1038/gt.2010.65 · Cited by 46

Endonucleases: tools to correct the dystrophin gene

Rousseau, J., Chapdelaine, P., Boisvert, S., de Almeida, L. P., Corbeil, J., Montpetit, A., & Tremblay, J. P. (2011) · The Journal of Gene Medicine · doi:10.1002/jgm.1611 · Cited by 46

Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells

Baum, C., Modlich, U., Göhring, G., & Schlegelberger, B. (2011) · Stem Cells · doi:10.1002/stem.716 · Cited by 44

Fate of Recombinant Adeno-Associated Viral Vector Genomes During DNA Double-Strand Break-Induced Gene Targeting in Human Cells

Gellhaus, K., Cornu, T. I., Heilbronn, R., & Cathomen, T. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.167 · Cited by 40

Corrigendum: Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

(2013) · Molecular Therapy · doi:10.1038/mt.2013.229 · Cited by 27

Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation

Benabdallah, B., Duval, A., Rousseau, J., Chapdelaine, P., Holmes, M. C., Haddad, É., Tremblay, J. P., & Beausejour, C. M. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.55 · Cited by 24

MegaTevs: single-chain dual nucleases for efficient gene disruption

Wolfs, J. M., DaSilva, M., Meister, S. E., Wang, X., Schild‐Poulter, C., & Edgell, D. R. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku573 · Cited by 24

Variable correction of Artemis deficiency by I-Sce1-meganuclease-assisted homologous recombination in murine hematopoietic stem cells

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