Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
Open accessAbstract
Homozygosity for the naturally occurring Delta32 deletion in the HIV co-receptor CCR5 confers resistance to HIV-1 infection. We generated an HIV-resistant genotype de novo using engineered zinc-finger nucleases (ZFNs) to disrupt endogenous CCR5. Transient expression of CCR5 ZFNs permanently and specifically disrupted approximately 50% of CCR5 alleles in a pool of primary human CD4(+) T cells. Genetic disruption of CCR5 provided robust, stable and heritable protection against HIV-1 infection in vitro and in vivo in a NOG model of HIV infection. HIV-1-infected mice engrafted with ZFN-modified CD4(+) T cells had lower viral loads and higher CD4(+) T-cell counts than mice engrafted with wild-type CD4(+) T cells, consistent with the potential to reconstitute immune function in individuals with HIV/AIDS by maintenance of an HIV-resistant CD4(+) T-cell population. Thus adoptive transfer of ex vivo expanded CCR5 ZFN-modified autologous CD4(+) T cells in HIV patients is an attractive approach for the treatment of HIV-1 infection.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Many studies have successfully disrupted CCR5 in various cell lines, primary T cells and hematopoietic stem cells, as well as in humanized mice.
“Since then, many studies have successfully disrupted CCR5 in various cell lines, primary T cells and hematopoietic stem cells (HSCs), as well as in humanized mice [30,31,32,38,39,40].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [30]
An adenoviral vector delivered ZFNs to activated CD4+ T cells in vitro, reaching a gene-disruption efficiency as high as 54% for CCR5 and 30%-34% for CXCR4.
“As a proof-of-concept delivery method, an adenoviral vector was used to deliver ZFNs to activated CD4+T cells in vitro, reaching a gene-disruption efficiency as high as 54% for CCR5 [30] and 30%–34% for CXCR4 [31,32].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [30]
Perez et al. found that with two mismatches, the cleavage activity for CCR5 is 10-fold higher than for CCR2.
“Perez et al. found that with two mismatches, the cleavage activity for CCR5 is 10-fold higher than for CCR2 [30].”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [30]
After expansion in vitro, adenovirus-ZFN transduced CD4+ T cells infused into humanized mice were able to establish resistance to HIV-1 infection.
“After expansion in vitro, the adenovirus-ZFN transduced CD4+ T cells were infused into humanized mice where they were able to establish resistance to HIV-1 infection.”
From Newer Gene Editing Technologies toward HIV Gene Therapy (2013) · cites this paper as [30]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Genome sequencing in microfabricated high-density picolitre reactors
Margulies, M., Egholm, M., Altman, W. E., Attiya, S., Bader, J. S., Bemben, L. A., Berka, J., Braverman, M. S., Chen, Y.-J., Chen, Z., et al. (2005) · Nature · doi:10.1038/nature03959 · Cited by 7,693 · see citing works
Two subsets of memory T lymphocytes with distinct homing potentials and effector functions
Sallusto, F., Lenig, D., Förster, R., Lipp, M., & Lanzavecchia, A. (1999) · Nature · doi:10.1038/44385 · Cited by 5,846 · see citing works
Identification of a major co-receptor for primary isolates of HIV-1
Deng, H., Liu, R., Ellmeier, W., Choe, S. S., Unutmaz, D., Burkhart, M. D., Marzio, P. D., Marmon, S., Sutton, R. E., Hill, C. M., et al. (1996) · Nature · doi:10.1038/381661a0 · Cited by 3,640 · see citing works
Liu, R., Paxton, W. A., Choe, S., Ceradini, D., Martin, S. R., Horuk, R., MacDonald, M. E., Stuhlmann, H., Koup, R. A., & Landau, N. R. (1996) · Cell · doi:10.1016/s0092-8674(00)80110-5 · Cited by 3,074 · see citing works
Samson, M., Libert, F., Doranz, B. J., Rucker, J. B., Liesnard, C., Farber, C., Saragosti, S., Lapouméroulie, C., Cognaux, J., Forceille, C., et al. (1996) · Nature · doi:10.1038/382722a0 · Cited by 2,885 · see citing works
CC CKR5: A RANTES, MIP-1α, MIP-1β Receptor as a Fusion Cofactor for Macrophage-Tropic HIV-1
Alkhatib, G., Combadière, C., Broder, C. C., Feng, Y., Kennedy, P. E., Murphy, P. M., & Berger, E. A. (1996) · Science · doi:10.1126/science.272.5270.1955 · Cited by 2,703 · see citing works
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
Urnov, F. D., Miller, J. C., Lee, Y.-L., Beausejour, C. M., Rock, J. M., Augustus, S., Jamieson, A. C., Porteus, M. H., Gregory, P. D., & Holmes, M. C. (2005) · Nature · doi:10.1038/nature03556 · Cited by 1,642 · see citing works
Self-citationThe role of a mutant CCR5 allele in HIV–1 transmission and disease progression
Huang, Y., Paxton, W. A., Wolinsky, S. M., Neumann, A. U., Zhang, L., Tian, H., Kang, S., Ceradini, D. J., Jin, Z., Yazdanbakhsh, K., et al. (1996) · Nature Medicine · doi:10.1038/nm1196-1240 · Cited by 1,359 · 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
Self-citationSchultz, L. B., Chehab, N. H., Malikzay, A., & Halazonetis, T. D. (2000) · The Journal of Cell Biology · doi:10.1083/jcb.151.7.1381 · Cited by 897 · 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
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 · see citing works
Self-citationEnhancing 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
Chimeric nucleases stimulate gene targeting in human cells
Porteus, M. H., & Baltimore, D. (2003) · Science · doi:10.1126/science.1078395 · Cited by 741 · see citing works
Qin, F. X.-F., An, D. S., Chen, I. S. Y., & Baltimore, D. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.232688199 · Cited by 701 · see citing works
Stimulation of Homologous Recombination through Targeted Cleavage by Chimeric Nucleases
Bibikova, M. V., Carroll, D., Segal, D. J., Trautman, J. K., Smith, J., Kim, Y., & Chandrasegaran, S. (2001) · Molecular and Cellular Biology · doi:10.1128/mcb.21.1.289-297.2001 · Cited by 614 · see citing works
Regulation and mechanisms of mammalian double-strand break repair
Valerie, K. C., & Povirk, L. F. (2003) · Oncogene · doi:10.1038/sj.onc.1206679 · Cited by 572 · see citing works
Gene transfer in humans using a conditionally replicating lentiviral vector
Levine, B. L., Humeau, L. M. P. F., Boyer, J., MacGregor, R.-R., Rebello, T., Lü, X., Binder, G. K., Slepushkin, V., Lemiale, F., Mascola, J. R., et al. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0608138103 · Cited by 505 · see citing works
Self-citationFätkenheuer, G., Pozniak, A., Johnson, M. A., Plettenberg, A., Staszewski, S., Hoepelman, A. I. M., Saag, M. S., Goebel, F. D., Rockstroh, J. K., Dezube, B. J., et al. (2005) · Nature Medicine · doi:10.1038/nm1319 · Cited by 495 · see citing works
Fok I dimerization is required for DNA cleavage
Bitinaite, J., Wah, D. A., Aggarwal, A. K., & Schildkraut, I. (1998) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.95.18.10570 · Cited by 490 · see citing works
Chromatin remodelling at a DNA double-strand break site in Saccharomyces cerevisiae
Tsukuda, T., Fleming, A. B., Nickoloff, J. A., & Osley, M. A. (2005) · Nature · doi:10.1038/nature04148 · Cited by 426 · see citing works
Smith, J. (2000) · Nucleic Acids Research · doi:10.1093/nar/28.17.3361 · Cited by 425 · see citing works
Targeted mutagenesis using zinc-finger nucleases in Arabidopsis
Lloyd, A., Plaisier, C. L., Carroll, D., & Drews, G. N. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0409339102 · Cited by 418 · see citing works
Genetic manipulation of genomes with rare-cutting endonucleases
Jasin, M. (1996) · Trends in Genetics · doi:10.1016/0168-9525(96)10019-6 · Cited by 391 · see citing works
Host-reactive CD8+ memory stem cells in graft-versus-host disease
Zhang, Y., Joe, G., Hexner, E., Zhu, J., & Emerson, S. G. (2005) · Nature Medicine · doi:10.1038/nm1326 · Cited by 369 · see citing works
Prevention of Vaginal SHIV Transmission in Rhesus Macaques Through Inhibition of CCR5
Lederman, M. M., Veazey, R. S., Offord, R. E., Mosier, D. E., Dufour, J., Mefford, M., Piatak, M., Lifson, J. D., Salkowitz, J. R., Rodriguez, B. A., et al. (2004) · Science · doi:10.1126/science.1099288 · Cited by 352 · see citing works
Mörner, A., Björndal, Å. S., Albert, J., KewalRamani, V. N., Littman, D. R., Inoue, R., Thorstensson, R., Fenyõ, É. M., & Björling, E. (1999) · Journal of Virology · doi:10.1128/jvi.73.3.2343-2349.1999 · Cited by 328 · see citing works
HIV-1 escape from a small molecule, CCR5-specific entry inhibitor does not involve CXCR4 use
Trkola, A., Kuhmann, S. E., Strizki, J., et al. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.012519099 · Cited by 303 · see citing works
Mutation detection using Surveyor™ nuclease
Qiu, P., Shandilya, H., D'Alessio, J. M., O’Connor, K. C., Durocher, J. A., & Gerard, G. F. (2004) · BioTechniques · doi:10.2144/04364pf01 · Cited by 299 · see citing works
Drug discovery with engineered zinc-finger proteins
Jamieson, A., Miller, J. C., & Pabo, C. (2003) · Nature reviews. Drug discovery · doi:10.1038/nrd1087 · Cited by 263 · see citing works
Self-citationLusso, P., Cocchi, F., Balotta, C., Markham, P. D., Louie, A. T., Farci, P., Pal, R., Gallo, R. C., & Reitz, M. S. (1995) · Journal of Virology · doi:10.1128/jvi.69.6.3712-3720.1995 · Cited by 256 · see citing works
Rossi, J. J., June, C. H., & Kohn, D. B. (2007) · Nature Biotechnology · doi:10.1038/nbt1367 · Cited by 233 · see citing works
Self-citationChromatin in Need of a Fix: Phosphorylation of H2AX Connects Chromatin to DNA Repair
Thiriet, C., & Hayes, J. J. (2005) · Molecular Cell · doi:10.1016/j.molcel.2005.05.008 · Cited by 205 · see citing works
Watanabe, S., Terashima, K., Ohta, S., et al. (2006) · Blood · doi:10.1182/blood-2006-04-017681 · Cited by 203 · see citing works
Kuhmann, S. E., Pugach, P., Kunstman, K., Taylor, J., Stanfield, R. L., Snyder, A., Strizki, J., Riley, J., Baroudy, B. M., Wilson, I. A., et al. (2004) · Journal of Virology · doi:10.1128/jvi.78.6.2790-2807.2004 · Cited by 194 · see citing works
Self-citationIsalan, M., Klug, A., & Choo, Y. (2001) · Nature Biotechnology · doi:10.1038/90264 · Cited by 191 · see citing works
Mosier, D. E., Picchio, G., Gulizia, R. J., et al. (1999) · Journal of Virology · doi:10.1128/jvi.73.5.3544-3550.1999 · Cited by 182 · see citing works
Levine, B. L., Bernstein, W. B., Aronson, N., Schlienger, K., Cotte, J., Perfetto, S. J., Humphries, M. J., Ratto‐Kim, S., Birx, D. L., Steffens, C. M., et al. (2002) · Nature Medicine · doi:10.1038/nm0102-47 · Cited by 169 · see citing works
Self-citationStable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates
An, D. S., Donahue, R. E., Kamata, M., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0705474104 · Cited by 150 · see citing works
Nilsson, M., Ljungberg, J., Richter, J., Kiefer, T., Magnusson, M., Lieber, A., Widegren, B., Karlsson, S., & Fan, X. (2004) · The Journal of Gene Medicine · doi:10.1002/jgm.543 · Cited by 129 · see citing works
Hung, C., Heyden, N. V., & Ratner, L. (1999) · Journal of Virology · doi:10.1128/jvi.73.10.8216-8226.1999 · Cited by 120 · see citing works
Steinberger, P., Andris‐Widhopf, J., Bühler, B., et al. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.2.805 · Cited by 113 · see citing works
Peters, W., Dupuis, M., & Charo, I. F. (2000) · The Journal of Immunology · doi:10.4049/jimmunol.165.12.7072 · Cited by 108 · see citing works
A detailed study of the substrate specificity of a chimeric restriction enzyme
Smith, J., Berg, J. M., & Chandrasegaran, S. (1999) · Nucleic Acids Research · doi:10.1093/nar/27.2.674 · Cited by 107 · see citing works
Bai, J., Gorantla, S., Banda, N. K., Cagnon, L., Rossi, J. J., & Akkina, R. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0038 · Cited by 99 · see citing works
CCR2 chemokine receptor and AIDS progression
Smith, M. W., Carrington, M. N., Winkler, C. A., Lomb, D. A., Dean, M. C., Huttley, G. A., & O’Brien, S. J. (1997) · Nature Medicine · doi:10.1038/nm1097-1052c · Cited by 99 · see citing works
Design of polyzinc finger peptides with structured linkers
Moore, M., Choo, Y., & Klug, A. (2001) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.98.4.1432 · Cited by 69 · see citing works
Schroers, R., Hildebrandt, Y., Hasenkamp, J., Glass, B., Lieber, A., Wulf, G., & Piesche, M. (2004) · Experimental Hematology · doi:10.1016/j.exphem.2004.03.010 · Cited by 60 · see citing works
Rapid, high-throughput engineering of sequence-specific zinc finger DNA-binding proteins
Isalan, M., & Choo, Y. (2001) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/s0076-6879(01)40444-7 · Cited by 37 · see citing works
Barassi, C., Soprana, E., Pastori, C., et al. (2005) · Journal of Virology · doi:10.1128/jvi.79.11.6848-6858.2005 · Cited by 31 · see citing works
Abad, J. L., González, M. A., del Real, G., Mira, E., Mañes, S., Serrano, F., & Bernad, A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00202-8 · Cited by 26 · see citing works
Flagged first, then by citations
Research works printed but not linked: 1
- [51]Morner A, et al. Primary human immunodeficiency virus type 2 (HIV-2) isolates, like HIV-1 isolates, frequently use CCR5 but show promiscuity in coreceptor usage. J. Virol. 1999;73:2343–2349. doi: 10.1128/jvi.73.3.2343-2349.1999. [DOI] [PMC free article] [PubMed] [Google Scholar] · DOI
Cited by
Sorted by citations
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
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
Gaj, T., Gersbach, C. A., & Barbas, C. F. (2013) · Trends in Biotechnology · doi:10.1016/j.tibtech.2013.04.004 · Cited by 3,820 · 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
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
Self-citationA 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
Self-citationTargeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
Cho, S. W., Kim, S., Kim, J. M., & Kim, J.-S. (2013) · Nature Biotechnology · doi:10.1038/nbt.2507 · Cited by 1,863 · see citing works
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 · see citing works
Pattanayak, V., Lin, S., Guilinger, J. P., Ma, E., Doudna, J. A., & Liu, D. R. (2013) · Nature Biotechnology · doi:10.1038/nbt.2673 · Cited by 1,487 · see citing works
Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018) · Science · doi:10.1126/science.aan4672 · Cited by 1,410 · see citing works
Gene Editing of <i>CCR5</i> in Autologous CD4 T Cells of Persons Infected with HIV
Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357 · see citing works
Self-citationEngineered T cells: the promise and challenges of cancer immunotherapy
Fesnak, A. D., June, C. H., & Levine, B. L. (2016) · Nature Reviews Cancer · doi:10.1038/nrc.2016.97 · Cited by 1,074 · see citing works
Self-citationMultiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition
Ren, J., Liu, X., Fang, C., Jiang, S., June, C. H., & Zhao, Y. (2017) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-16-1300 · Cited by 942 · see citing works
Self-citationGuilinger, J. P., Thompson, D. B., & Liu, D. R. (2014) · Nature Biotechnology · doi:10.1038/nbt.2909 · Cited by 836 · see citing works
Soldner, F., Laganière, J., Cheng, A. W., Hockemeyer, D., Gao, Q., Alagappan, R., Khurana, V., Golbe, L. I., Myers, R. H., Lindquist, S., et al. (2011) · Cell · doi:10.1016/j.cell.2011.06.019 · Cited by 728 · see citing works
Self-citationThe Discovery of Zinc Fingers and Their Applications in Gene Regulation and Genome Manipulation
Klug, A. (2010) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-010909-095056 · Cited by 710 · see citing works
A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
Mussolino, C., Morbitzer, R., Lütge, F., Dannemann, N., Lahaye, T., & Cathomen, T. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr597 · Cited by 714 · see citing works
Holt, N., Wang, J., Kim, K., Friedman, G., Wang, X., Taupin, V., Crooks, G. M., Kohn, D. B., Gregory, P. D., Holmes, M. C., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1663 · Cited by 677 · see citing works
Self-citationWilen, C. B., Tilton, J. C., & Doms, R. W. (2012) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a006866 · Cited by 654 · 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
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 · see citing works
Self-citationAn 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 · see citing works
Self-citationEfficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9
Mandal, P. K., Ferreira, L. M. R., Collins, R., Meissner, T. B., Boutwell, C. L., Friesen, M., Vrbanac, V., Garrison, B. S., Stortchevoi, A., Bryder, D., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.10.004 · Cited by 475 · see citing works
Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly
Kim, H. J., Lee, H. J., Kim, H., Cho, S. W., & Kim, J.-S. (2009) · Genome Research · doi:10.1101/gr.089417.108 · Cited by 466 · see citing works
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
Xu, L., Wang, J., Liu, Y., Xie, L., Su, B., Mou, D., Wang, L., Liu, T., Wang, X., Zhang, B., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1817426 · Cited by 454 · see citing works
Self-citationRevealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection
Pattanayak, V., Ramirez, C. L., Joung, J. K., & Liu, D. R. (2011) · Nature Methods · doi:10.1038/nmeth.1670 · Cited by 453 · see citing works
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
Doyon, Y., Vo, T. D., Mendel, M. C., Greenberg, S. G., Wang, J., Xia, D. F., Miller, J. C., Urnov, F. D., Gregory, P. D., & Holmes, M. C. (2011) · Nature Methods · doi:10.1038/nmeth.1539 · Cited by 402 · see citing works
Self-citationZischewski, J., Fischer, R., & Bortesi, L. (2017) · Biotechnology Advances · doi:10.1016/j.biotechadv.2016.12.003 · Cited by 323 · see citing works
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 · see citing works
In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Sharma, R., Anguela, X. M., Doyon, Y., Wechsler, T., DeKelver, R. C., Sproul, S., Paschon, D. E., Miller, J. C., Davidson, R. J., Shivak, D., et al. (2015) · Blood · doi:10.1182/blood-2014-12-615492 · Cited by 297 · see citing works
Self-citationWang, J., Exline, C. M., DeClercq, J. J., Llewellyn, G. N., Hayward, S. B., Li, P. W.-L., Shivak, D. A., Surosky, R. T., Gregory, P. D., Holmes, M. C., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3408 · Cited by 261 · see citing works
Self-citationA New Class of Medicines through DNA Editing
Porteus, M. H. (2019) · New England Journal of Medicine · doi:10.1056/nejmra1800729 · Cited by 229 · see citing works
Subburaj, S., Chung, S. J., Lee, C., Ryu, S.-M., Kim, D. H., Kim, J.-S., Bae, S., & Lee, G.-J. (2016) · Plant Cell Reports · doi:10.1007/s00299-016-1937-7 · Cited by 237 · see citing works
Self-citationIntegration-deficient Lentiviral Vectors: A Slow Coming of Age
Wanisch, K., & Yáñez-Muñoz, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.122 · Cited by 222 · see citing works
Guo, J., Gaj, T., & Barbas, C. F. (2010) · Journal of Molecular Biology · doi:10.1016/j.jmb.2010.04.060 · Cited by 206 · see citing works
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease
Hoban, M. D., Orkin, S. H., & Bauer, D. E. (2016) · Blood · doi:10.1182/blood-2015-09-618587 · Cited by 194 · see citing works
Hultquist, J. F., Schumann, K., Woo, J. M., Manganaro, L., McGregor, M. J., Doudna, J., Simon, V., Krogan, N. J., & Marson, A. (2016) · Cell Reports · doi:10.1016/j.celrep.2016.09.080 · Cited by 170 · see citing works
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 · see citing works
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 · see citing works
Self-citationHIV reservoirs: pathogenesis and obstacles to viral eradication and cure
Chun, T.-W., & Fauci, A. S. (2012) · AIDS · doi:10.1097/qad.0b013e328353f3f1 · Cited by 175 · see citing works
Qu, X., Wang, P., Ding, D., Li, L., Wang, H., Ma, L., Zhou, X., Liu, S., Lin, S., Wang, X., et al. (2013) · Nucleic Acids Research · doi:10.1093/nar/gkt571 · Cited by 155 · see citing works
Self-citationEngineering 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 · see citing works
Wang, J., DeClercq, J. J., Hayward, S. B., Li, P. W.-L., Shivak, D. A., Gregory, P. D., Lee, G., & Holmes, M. C. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkv1121 · Cited by 128 · see citing works
Self-citationMaier, D. A., Brennan, A. L., Jiang, S., Binder-Scholl, G. K., Lee, G., Plesa, G., Zheng, Z., Cotte, J., Carpenito, C., Wood, T., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.172 · Cited by 126 · see citing works
Self-citationTools for experimental and computational analyses of off-target editing by programmable nucleases
Bao, X. R., Pan, Y., Lee, C. M., Davis, T. H., & Bao, G. (2021) · Nature Protocols · doi:10.1038/s41596-020-00431-y · Cited by 106 · see citing works
Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases
Liu, P., Chan, E. M., Cost, G. J., Zhang, L., Wang, J., Miller, J. C., Guschin, D. Y., Reik, A., Holmes, M. C., Mott, J. E., et al. (2010) · Biotechnology and Bioengineering · doi:10.1002/bit.22654 · Cited by 107 · see citing works
Self-citationZinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment
Yuan, J., Wang, J., Crain, K., Fearns, C., Kim, K. A., Hua, K. L., Gregory, P. D., Holmes, M. C., & Torbett, B. E. (2012) · Molecular Therapy · doi:10.1038/mt.2011.310 · Cited by 105 · see citing works
Self-citationAntibody glycoengineering strategies in mammalian cells
Wang, Q., Chung, C., Chough, S., & Betenbaugh, M. J. (2018) · Biotechnology and Bioengineering · doi:10.1002/bit.26567 · Cited by 94 · see citing works
Newer Gene Editing Technologies toward HIV Gene Therapy
Manjunath, N., Yi, G., Dang, Y., & Shankar, P. (2013) · Viruses · doi:10.3390/v5112748 · Cited by 77 · see citing works
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 · see citing works
Adeno-associated Virus: A Key to the Human Genome?
Henckaerts, E., & Linden, R. M. (2010) · Future Virology · doi:10.2217/fvl.10.48 · Cited by 71 · see citing works
Towards a new era in medicine: therapeutic genome editing
Porteus, M. H. (2015) · Genome Biology · doi:10.1186/s13059-015-0859-y · Cited by 69 · see citing works
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 · see citing works
Targeted gene disruption to cure HIV
Stone, D., Kiem, H.-P., & Jerome, K. R. (2013) · Current Opinion in HIV and AIDS · doi:10.1097/coh.0b013e32835f736c · Cited by 55 · see citing works
A novel therapeutic molecule against HTLV-1 infection targeting provirus
Tanaka, A., Takeda, S., Kariya, R., Matsuda, K., Urano, E., Okada, S., & Komano, J. (2013) · Leukemia · doi:10.1038/leu.2013.46 · Cited by 46 · see citing works
Schiffer, J. T., Swan, D. A., Stone, D., & Jerome, K. R. (2013) · PLoS Computational Biology · doi:10.1371/journal.pcbi.1003131 · Cited by 44 · see citing works
Hofer, U., Henley, J. E., Exline, C. M., Mulhern, O., Lopez, E., & Cannon, P. M. (2013) · Journal of Infectious Diseases · doi:10.1093/infdis/jit382 · Cited by 43 · see citing works
Lei, Y., Lee, C.-L., Joo, K.-I., Zarzar, J., Liu, Y., Dai, B., Fox, V., & Wang, P. (2011) · Molecular Therapy · doi:10.1038/mt.2011.12 · Cited by 42 · see citing works
Wayengera, M. (2011) · Theoretical Biology and Medical Modelling · doi:10.1186/1742-4682-8-26 · Cited by 21 · see citing works
Genome Editing for the β-Hemoglobinopathies
Porteus, M. H. (2017) · Advances in Experimental Medicine and Biology · doi:10.1007/978-1-4939-7299-9_8 · Cited by 8 · see citing works
- Authors
- Perez, E. E., Wang, J., Miller, J. C., Jouvenot, Y., Kim, K. A., Liu, O., Wang, N., Lee, G., Bartsevich, V. V., Lee, Y.-L., Guschin, D. Y., Rupniewski, I., Waite, A. J., Carpenito, C., Carroll, R. G., S Orange, J., Urnov, F. D., Rebar, E. J., Ando, D., Gregory, P. D., Riley, J. L., Holmes, M. C., & June, C. H.
- Affiliations
- Sangamo BioSciences (United States) (US)
- Abramson Cancer Center (US)
- Corresponding author
- June, Carl H
- ORCID
- Venue
- Nature Biotechnology
- Published
- 2008-06-29
- Volume
- 26
- Issue
- 7
- Pages
- 808-816
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- PMC
- PMC3422503
- OpenAlex
- W2110260408
- Cited by
- 999
- References
- 51
- Citations by year
- 2026: 5 · 2025: 24 · 2024: 21 · 2023: 22 · 2022: 24 · 2021: 45 · 2020: 53 · 2019: 36 · 2018: 57 · 2017: 63 · 2016: 86 · 2015: 92 · 2014: 100 · 2013: 97 · 2012: 87
- FWCI
- 25.34
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- University of Pennsylvania
- National Heart, Lung, and Blood Institute (T32HL007775)
- National Institute of Allergy and Infectious Diseases (K08AI062468, U19AI082628)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Immunology and Microbiology › Virology › HIV Research and Treatment
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- HIV-1 resistance, CCR5, zinc finger nucleases, genome editing, CD4+ T cells, CCR5Δ32, HIV co-receptor, adoptive T cell therapy, HIV-1 infection, viral load, immune reconstitution
- MeSH terms
- Animals, Cells, Cultured, Chromosome Mapping, Deoxyribonucleases, Genetic Engineering, Humans, Immunity, Innate, CD4-Positive T-Lymphocytes, HIV Infections, Zinc Fingers, Treatment Outcome, Adoptive Transfer, Mice
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
