Ex vivo gene transfer and correction for cell-based therapies

Fate Mapping Analysis Reveals That Adult Microglia Derive from Primitive Macrophages

Ginhoux, F., Greter, M., Leboeuf, M., Nandi, S., See, P., Gokhan, S., Mehler, M. F., Conway, S. J., Ng, L. G., Stanley, E. R., et al. (2010) · Science · doi:10.1126/science.1194637 · Cited by 5,175

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector

Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903

The DNA Damage Response: Making It Safe to Play with Knives

Ciccia, A., & Elledge, S. J. (2010) · Molecular Cell · doi:10.1016/j.molcel.2010.09.019 · Cited by 4,368

Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study

Le Blanc, K., Frassoni, F., Ball, L., Locatelli, F., Roelofs, H., Lewis, I., Lanino, E., Sundberg, B., Bernardo, M. E., Remberger, M., et al. (2008) · The Lancet · doi:10.1016/s0140-6736(08)60690-x · Cited by 2,695

Hematopoietic Stem-Cell Transplantation

Copelan, E. A. (2006) · New England Journal of Medicine · doi:10.1056/nejmra052638 · Cited by 2,522

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

Natural Innate and Adaptive Immunity to Cancer

Vesely, M. D., Kershaw, M. H., Schreiber, R. D., & Smyth, M. J. (2011) · Annual Review of Immunology · doi:10.1146/annurev-immunol-031210-101324 · Cited by 2,101

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

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

Christian, M., Cermak, T., Doyle, E. L., Schmidt, C., Zhang, F., Hummel, A., Bogdanove, A. J., & Voytas, D. F. (2010) · Genetics · doi:10.1534/genetics.110.120717 · Cited by 1,869

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Hacein-Bey-Abina, S., Garrigue, A., Wang, G. P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35700 · Cited by 1,854

Local self-renewal can sustain CNS microglia maintenance and function throughout adult life

Ajami, B., Bennett, J. L., Krieger, C., Tetzlaff, W., & Rossi, F. M. V. (2007) · Nature Neuroscience · doi:10.1038/nn2014 · Cited by 1,643

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 1,641

Mesenchymal Stem Cells: Revisiting History, Concepts, and Assays

Bianco, P., Robey, P. G., & Simmons, P. J. (2008) · Cell Stem Cell · doi:10.1016/j.stem.2008.03.002 · Cited by 1,621

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

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

Molecular Reconstruction of Sleeping Beauty, a Tc1-like Transposon from Fish, and Its Transposition in Human Cells

Ivics, Z., Hackett, P. B., Plasterk, R. H., & Izsvák, Z. (1997) · Cell · doi:10.1016/s0092-8674(00)80436-5 · Cited by 1,430

Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration

Wu, X., Li, Y., Crise, B., & Burgess, S. M. (2003) · Science · doi:10.1126/science.1083413 · Cited by 1,305

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

Howe, S. J., Mansour, M. R., Schwarzwaelder, K., Bartholomae, C., Hubank, M., Kempski, H., Brugman, M. H., Pike-Overzet, K., Chatters, S. J., de Ridder, D., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35798 · Cited by 1,286

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K., et al. (2010) · Nature · doi:10.1038/nature09328 · Cited by 1,276

Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma

Pule, M. A., Savoldo, B., Myers, G. D., Rossig, C., Russell, H. V., Dotti, G., Huls, M. H., Liu, E., Gee, A. P., Mei, Z., et al. (2008) · Nature Medicine · doi:10.1038/nm.1882 · Cited by 1,195

Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration

Rama, P., Matuska, S., Paganoni, G., Spinelli, A., De Luca, M., & Pellegrini, G. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa0905955 · Cited by 1,141

Microglia in the adult brain arise from Ly-6ChiCCR2+ monocytes only under defined host conditions

Mildner, A., Schmidt, H., Nitsche, M., Merkler, D., Hanisch, U.-K., Mack, M., Heikenwalder, M., Brück, W., Priller, J., & Prinz, M. (2007) · Nature Neuroscience · doi:10.1038/nn2015 · Cited by 1,089

The Primary Immunodeficiencies

Rosen, F. S., Cooper, M. D., & Wedgwood, R. J. P. (1984) · New England Journal of Medicine · doi:10.1056/nejm198408023110506 · Cited by 1,077

Removal of homeostatic cytokine sinks by lymphodepletion enhances the efficacy of adoptively transferred tumor-specific CD8 + T cells

Gattinoni, L., Finkelstein, S. E., Klebanoff, C. A., Antony, P. A., Palmer, D. C., Spiess, P. J., Hwang, L. N., Yu, Z., Wrzesinski, C., Heimann, D. M., et al. (2005) · The Journal of Experimental Medicine · doi:10.1084/jem.20050732 · Cited by 1,073

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

Hockemeyer, D. F., Soldner, F., Beard, C., Gao, Q., Mitalipova, M. M., DeKelver, R. C., Katibah, G. E., Amora, R., Boydston, E. A., Zeitler, B., et al. (2009) · Nature Biotechnology · doi:10.1038/nbt.1562 · Cited by 1,070

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

Ott, M. G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A., Kunkel, H., et al. (2006) · Nature Medicine · doi:10.1038/nm1393 · Cited by 1,051

Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells

Boitano, A. E., Wang, J., Romeo, R., Bouchez, L. C., Parker, A. E., Sutton, S. E., Walker, J. R., Flaveny, C. A., Perdew, G. H., Denison, M. S., et al. (2010) · Science · doi:10.1126/science.1191536 · Cited by 1,047

Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis

North, T. E., Goessling, W., Walkley, C. R., Lengerke, C., Kopani, K. R., Lord, A. M., Weber, G. J., Bowman, T. V., Jang, I.-H., Grosser, T., et al. (2007) · Nature · doi:10.1038/nature05883 · Cited by 1,029

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Aiuti, A., Cattaneo, F., Galimberti, S., Benninghoff, U., Cassani, B., Callegaro, L., Scaramuzza, S., Andolfi, G., Mirolo, M., Brigida, I., et al. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa0805817 · Cited by 1,007

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

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., et al. (2008) · Nature Biotechnology · doi:10.1038/nbt1410 · Cited by 999

Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences

Mitchell, R. S., Beitzel, B. F., Schroder, A. R. W., Shinn, P., Chen, H., Berry, C. C., Ecker, J. R., & Bushman, F. D. (2004) · PLoS Biology · doi:10.1371/journal.pbio.0020234 · Cited by 976

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) · Science · doi:10.1126/science.1070104 · Cited by 975

Acquisition of full effector function in vitro paradoxically impairs the in vivo antitumor efficacy of adoptively transferred CD8+ T cells

Gattinoni, L. (2005) · Journal of Clinical Investigation · doi:10.1172/jci24480 · Cited by 958

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Mátés, L., Chuah, M. K. L., Belay, E., Jerchow, B., Manoj, N., Acosta-Sanchez, A., Grzela, D. P., Schmitt, A., Becker, K., Matrai, J., et al. (2009) · Nature Genetics · doi:10.1038/ng.343 · Cited by 948

Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges

Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893

Mesenchymal Stem Cells: Mechanisms of Inflammation

Singer, N. G., & Caplan, A. I. (2011) · Annual Review of Pathology: Mechanisms of Disease · doi:10.1146/annurev-pathol-011110-130230 · Cited by 851

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

Adoptive transfer of effector CD8+ T cells derived from central memory cells establishes persistent T cell memory in primates

Berger, C., Jensen, M. C., Lansdorp, P. M., Gough, M., Elliott, C., & Riddell, S. R. (2008) · Journal of Clinical Investigation · doi:10.1172/jci32103 · Cited by 822

Current prospects for RNA interference-based therapies

Davidson, B. L., & McCray, P. B. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2968 · Cited by 789

Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism

Pluchino, S., Zanotti, L., Rossi, B., Brambilla, E., Ottoboni, L., Salani, G., Martinello, M., Cattalini, A., Bergami, A., Furlan, R., et al. (2005) · Nature · doi:10.1038/nature03889 · Cited by 768

Chimeric nucleases stimulate gene targeting in human cells

Porteus, M. H., & Baltimore, D. (2003) · Science · doi:10.1126/science.1078395 · Cited by 741

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

Stein, S., Ott, M. G., Schultze-Strasser, S., Jauch, A., Burwinkel, B., Kinner, A., Schmidt, M., Krämer, A., Schwäble, J., Glimm, H., et al. (2010) · Nature Medicine · doi:10.1038/nm.2088 · Cited by 716

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Ponzoni, M., Bartholomae, C. C., Sergi, L. S., Benedicenti, F., Ambrosi, A., Serio, C. D., et al. (2006) · Nature Biotechnology · doi:10.1038/nbt1216 · Cited by 711

Pluripotency and Cellular Reprogramming: Facts, Hypotheses, Unresolved Issues

Hanna, J. H., Saha, K., & Jaenisch, R. (2010) · Cell · doi:10.1016/j.cell.2010.10.008 · Cited by 698

Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

Tedesco, F. S., Dellavalle, A., Diaz-Manera, J., Messina, G., & Cossu, G. (2010) · Journal of Clinical Investigation · doi:10.1172/jci40373 · Cited by 677

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

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

HUMAN T CELL RESPONSES AGAINST MELANOMA

Boon, T., Coulie, P. G., den Eynde, B. J. V., & van der Bruggen, P. (2005) · Annual Review of Immunology · doi:10.1146/annurev.immunol.24.021605.090733 · Cited by 671

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

Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 666

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Hauer, J., Lim, A., Picard, C., Wang, G. P., Berry, C. C., Martinache, C., Rieux-Laucat, F., Latour, S., Belohradsky, B. H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000164 · Cited by 639

Targeting gene-modified hematopoietic cells to the central nervous system: use of green fluorescent protein uncovers microglial engraftment

Priller, J., Flügel, A., Wehner, T., Boentert, M., Haas, C. A., Prinz, M., Fernández-Klett, F., Prass, K., Bechmann, I., de Boer, B. A., et al. (2001) · Nature Medicine · doi:10.1038/nm1201-1356 · Cited by 637

Stem cells in human neurodegenerative disorders — time for clinical translation?

Lindvall, O., & Kokaia, Z. (2010) · Journal of Clinical Investigation · doi:10.1172/jci40543 · Cited by 614

Correction of sickle cell disease in transgenic mouse models by gene therapy

Pawliuk, R., Westerman, K. A., Fabry, M. E., Payen, E., Tighe, R., Bouhassira, E. E., Acharya, S. A., Ellis, J., London, I. M., Eaves, C. J., et al. (2001) · Science · doi:10.1126/science.1065806 · Cited by 579 · cited 1×

Repair of Tissues by Adult Stem/Progenitor Cells (MSCs): Controversies, Myths, and Changing Paradigms

Prockop, D. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.62 · Cited by 577

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

Brown, B. D., Gentner, B., Cantore, A., Colleoni, S., Amendola, M., Zingale, A., Baccarini, A., Lazzari, G., Galli, C., & Naldini, L. (2007) · Nature Biotechnology · doi:10.1038/nbt1372 · Cited by 572

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37630 · Cited by 571

Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin

May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K. M. L., Luzzatto, L., & Sadelain, M. (2000) · Nature · doi:10.1038/35017565 · Cited by 567

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

Boztug, K., Schmidt, M., Schwarzer, A., Banerjee, P. P., Díez, I. A., Dewey, R. A., Böhm, M., Nowrouzi, A., Ball, C. R., Glimm, H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1003548 · Cited by 532

Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)

Sander, J. D., Dahlborg, E. J., Goodwin, M. J., Cade, L., Zhang, F., Cifuentes, D., Curtin, S. J., Blackburn, J. S., Thibodeau-Beganny, S., Qi, Y., et al. (2010) · Nature Methods · doi:10.1038/nmeth.1542 · Cited by 523

Regulatory T-cell immunotherapy for tolerance to self antigens and alloantigens in humans

Roncarolo, M., & Battaglia, M. (2007) · Nature reviews. Immunology · doi:10.1038/nri2138 · Cited by 522

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

Zou, J., Maeder, M. L., Mali, P., Pruett‐Miller, S. M., Thibodeau-Beganny, S., Chou, B.-K., Chen, G., Ye, Z., Park, I., Daley, G. Q., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.023 · Cited by 513

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I–II study

Ciceri, F., Bonini, C., Stanghellini, M. T. L., Bondanza, A., Traversari, C., Salomoni, M., Turchetto, L., Colombi, S., Bernardi, M., Peccatori, J., et al. (2009) · The Lancet Oncology · doi:10.1016/s1470-2045(09)70074-9 · Cited by 488

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

Brown, B. D., Venneri, M. A., Zingale, A., Sergi, L. S., & Naldini, L. (2006) · Nature Medicine · doi:10.1038/nm1398 · Cited by 484

Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy

Bendle, G. M., Linnemann, C., Hooijkaas, A. I., Bies, L., de Witte, M. A., Jorritsma, A., Kaiser, A. D. M., Pouw, N., Debets, R., Kieback, E., et al. (2010) · Nature Medicine · doi:10.1038/nm.2128 · Cited by 448

Cytokine Signals Are Sufficient for HIV-1 Infection of Resting Human T Lymphocytes

Unutmaz, D., KewalRamani, V. N., Marmon, S., & Littman, D. R. (1999) · The Journal of Experimental Medicine · doi:10.1084/jem.189.11.1735 · Cited by 427

Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?

Gennery, A. R., Slatter, M. A., Grandin, L., Taupin, P., Cant, A. J., Veys, P., Amrolia, P. J., Gaspar, H. B., Davies, E. G., Friedrich, W., et al. (2010) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2010.06.015 · Cited by 427

Genome-wide analysis of retroviral DNA integration

Bushman, F., Lewinski, M., Ciuffi, A., Barr, S., Leipzig, J., Hannenhalli, S., & Hoffmann, C. (2005) · Nature Reviews Microbiology · doi:10.1038/nrmicro1263 · Cited by 422

Primary immunodeficiencies

Notarangelo, L. D. (2010) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2009.07.053 · Cited by 417

Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy

Silva, G., Poirot, L., Galetto, R., Smith, J., Montoya, G., Duchateau, P., & Paques, F. (2011) · Current Gene Therapy · doi:10.2174/156652311794520111 · Cited by 410

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M. H., Schambach, A., Charrier, S., Galy, A., Thrasher, A. J., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.179 · Cited by 387

Silencing and Variegation of Gammaretrovirus and Lentivirus Vectors

Ellis, J. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.1241 · Cited by 379

RNA-Based Gene Therapy for HIV with Lentiviral Vector–Modified CD34 + Cells in Patients Undergoing Transplantation for AIDS-Related Lymphoma

DiGiusto, D. L., Krishnan, A., Li, L., Li, H., Li, S., Rao, A., Mi, S., Yam, P., Stinson, S., Kalos, M., et al. (2010) · Science Translational Medicine · doi:10.1126/scitranslmed.3000931 · Cited by 374

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

Brown, B. D., & Naldini, L. (2009) · Nature Reviews Genetics · doi:10.1038/nrg2628 · Cited by 336

Neural stem cell systems: physiological players or in vitro entities?

Conti, L., & Cattaneo, E. (2010) · Nature Reviews Neuroscience · doi:10.1038/nrn2761 · Cited by 320

Stable knockdown of microRNA in vivo by lentiviral vectors

Gentner, B., Schira, G., Giustacchini, A., Amendola, M., Brown, B. D., Ponzoni, M., & Naldini, L. (2008) · Nature Methods · doi:10.1038/nmeth.1277 · Cited by 309

Tumor-Targeted Interferon-α Delivery by Tie2-Expressing Monocytes Inhibits Tumor Growth and Metastasis

De Palma, M., Mazzieri, R., Politi, L. S., Pucci, F., Zonari, E., Sitia, G., Mazzoleni, S., Moi, D., Venneri, M. A., Indraccolo, S., et al. (2008) · Cancer Cell · doi:10.1016/j.ccr.2008.09.004 · Cited by 301

Pleiotrophin regulates the expansion and regeneration of hematopoietic stem cells

Himburg, H. A., Muramoto, G. G., Daher, P., Meadows, S. K., Russell, J. L., Doan, P. L., Chi, J., Salter, A. B., Lento, W. E., Reya, T., et al. (2010) · Nature Medicine · doi:10.1038/nm.2119 · Cited by 298

Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease

Lee, J. P., Jeyakumar, M., Gonzalez, R., Takahashi, H., Lee, P. J., Baek, R. C., Clark, D. B., Rose, H., Fu, G., Clarke, J., et al. (2007) · Nature Medicine · doi:10.1038/nm1548 · Cited by 287

Comparison of human cord blood engraftment between immunocompromised mouse strains

McDermott, S. P., Eppert, K., Lechman, E. R., Doedens, M., & Dick, J. E. (2010) · Blood · doi:10.1182/blood-2010-02-271841 · Cited by 284

Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking

Kustikova, O., Fehse, B., Modlich, U., Yang, M., Düllmann, J., Kamino, K., von Neuhoff, N., Schlegelberger, B., Li, Z., & Baum, C. (2005) · Science · doi:10.1126/science.1105063 · Cited by 283

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

Biffi, A., De Palma, M., Quattrini, A., Del Carro, U., Amadio, S., Visigalli, I., Sessa, M., Fasano, S., Brambilla, R., Marchesini, S., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci200419205 · Cited by 280

Cell culture assays reveal the importance of retroviral vector design for insertional genotoxicity

Modlich, U., Bohne, J., Schmidt, M., von Kalle, C., Knöss, S., Schambach, A., & Baum, C. (2006) · Blood · doi:10.1182/blood-2005-08-024976 · Cited by 278

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., & Baum, C. (2008) · Molecular Therapy · doi:10.1038/mt.2008.5 · Cited by 271

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

Biffi, A., De Palma, M., Quattrini, A., Del Carro, U., Amadio, S., Visigalli, I., Sessa, M., Fasano, S., Brambilla, R., Marchesini, S., et al. (2004) · Journal of Clinical Investigation · doi:10.1172/jci19205 · Cited by 261

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

Deichmann, A., Hacein‐Bey‐Abina, S., Schmidt, M., Garrigue, A., Brugman, M. H., Hu, J., Glimm, H., Gyapay, G., Prum, B., Fraser, C. C., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci31659 · Cited by 260

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Aiuti, A., Cassani, B., Andolfi, G., Mirolo, M., Biasco, L., Recchia, A., Urbinati, F., Valacca, C., Scaramuzza, S., Aker, M., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci31666 · Cited by 257

Expansion of human cord blood CD34+CD38−cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: dissociation of SRC phenotype and function

Dorrell, C., Gan, O. I., PEREIRA, D. S., Hawley, R. G., & Dick, J. E. (2000) · Blood · doi:10.1182/blood.v95.1.102 · Cited by 250

Cardiogenesis and the Complex Biology of Regenerative Cardiovascular Medicine

Chien, K. R., Domian, I. J., & Parker, K. K. (2008) · Science · doi:10.1126/science.1163267 · Cited by 249

Angiopoietin-like 5 and IGFBP2 stimulate ex vivo expansion of human cord blood hematopoietic stem cells as assayed by NOD/SCID transplantation

Zhang, C. C., Kaba, M., Iizuka, S., Huynh, H., & Lodish, H. F. (2008) · Blood · doi:10.1182/blood-2007-11-122119 · Cited by 246

Genetic therapies against HIV

Rossi, J. J., June, C. H., & Kohn, D. B. (2007) · Nature Biotechnology · doi:10.1038/nbt1367 · Cited by 233

Progress and prospects: Zinc-finger nucleases as gene therapy agents

Carroll, D. (2008) · Gene Therapy · doi:10.1038/gt.2008.145 · Cited by 233

Mixed T cell receptor dimers harbor potentially harmful neoreactivity

van Loenen, M. M., de Boer, R., Amir, A. L., Hagedoorn, R. S., Volbeda, G. L., Willemze, R., van Rood, J. J., Falkenburg, J. H. F., & Heemskerk, M. H. M. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1005802107 · Cited by 231

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

Biffi, A., Capotondo, A., Fasano, S., Carro, U. D., Marchesini, S., Azuma, H., Malaguti, M. C., Amadio, S., Brambilla, R., Grompe, M., et al. (2006) · Journal of Clinical Investigation · doi:10.1172/jci28873 · Cited by 223

Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning

Gaspar, H. B., Bjorkegren, E., Parsley, K., Gilmour, K. C., King, D., Sinclair, J. C., Zhang, F., Giannakopoulos, A., Adams, S. P., Fairbanks, L. D., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.06.007 · Cited by 217

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

Biffi, A., Bartolomae, C. C., Cesana, D., Cartier, N., Aubourg, P., Ranzani, M., Cesani, M., Benedicenti, F., Plati, T., Rubagotti, E., et al. (2011) · Blood · doi:10.1182/blood-2010-09-306761 · Cited by 216

Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

Gentner, B., Visigalli, I., Hiramatsu, H., Lechman, E. R., Ungari, S., Giustacchini, A., Schira, G., Amendola, M., Quattrini, A., Martino, S., et al. (2010) · Science Translational Medicine · doi:10.1126/scitranslmed.3001522 · Cited by 210

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

Krivit, W., Sung, J. H., Shapiro, E., & Lockman, L. A. (1995) · Cell Transplantation · doi:10.1016/0963-6897(95)00021-o · Cited by 207

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

Schwarzwaelder, K., Howe, S. J., Schmidt, M., Brugman, M. H., Deichmann, A., Glimm, H., Schmidt, S. K., Prinz, C., Wissler, M., King, D., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci31661 · Cited by 203

Engineering lymphocyte subsets: tools, trials and tribulations

June, C. H., Blazar, B. R., & Riley, J. L. (2009) · Nature reviews. Immunology · doi:10.1038/nri2635 · Cited by 199

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

Kang, E. M., Choi, U., Theobald, N., Linton, G. F., Priel, D. A. L., Kuhns, D. B., & Malech, H. L. (2009) · Blood · doi:10.1182/blood-2009-05-222760 · Cited by 193

Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells

Recchia, A., Bonini, C., Magnani, Z. I., Urbinati, F., Sartori, D., Muraro, S., Tagliafico, E., Bondanza, A., Stanghellini, M. T. L., Bernardi, M., et al. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0507496103 · Cited by 187

Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis

Modlich, U. (2005) · Blood · doi:10.1182/blood-2004-11-4535 · Cited by 186

Comprehensive genomic access to vector integration in clinical gene therapy

Gabriel, R., Eckenberg, R., Paruzynski, A., Bartholomae, C. C., Nowrouzi, A., Arens, A., Howe, S. J., Recchia, A., Cattoglio, C., Wang, W., et al. (2009) · Nature Medicine · doi:10.1038/nm.2057 · Cited by 185

Improved Expression and Reactivity of Transduced Tumor-Specific TCRs in Human Lymphocytes by Specific Silencing of Endogenous TCR

Okamoto, S., Mineno, J., Ikeda, H., Fujiwara, H., Yasukawa, M., Shiku, H., & Kato, I. (2009) · Cancer Research · doi:10.1158/0008-5472.can-09-1450 · Cited by 185

Safety of retroviral gene marking with a truncated NGF receptor

Bonini, C., Grez, M., Traversari, C., Ciceri, F., Marktel, S., Ferrari, G., Dinauer, M., Sadat, M. A., Aiuti, A., Deola, S., et al. (2003) · Nature Medicine · doi:10.1038/nm0403-367 · Cited by 182

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model

Visigalli, I., Delai, S., Politi, L. S., Domenico, C. D., Cerri, F., Mrak, E., d’Isa, R., Ungaro, D., Stok, M., Sanvito, F., et al. (2010) · Blood · doi:10.1182/blood-2010-04-278234 · Cited by 179

Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells

Palma, M. D., Montini, E., de Sio, F. R. S., Benedicenti, F., Gentile, A., Médico, E., & Naldini, L. (2004) · Blood · doi:10.1182/blood-2004-03-0798 · Cited by 172

Regeneration Next: Toward Heart Stem Cell Therapeutics

Hansson, E. M., Lindsay, M. E., & Chien, K. R. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.09.004 · Cited by 170

Engineering human hematopoietic stem/progenitor cells to produce a broadly neutralizing anti-HIV antibody after in vitro maturation to human B lymphocytes.

Luo, X., Maarschalk, E., O’Connell, R. M., Wang, P., Yang, L., & Baltimore, D. (2008) · Blood · doi:10.1182/blood-2008-09-177139 · Cited by 168

20 years of gene therapy for SCID

Fischer, A., Hacein‐Bey‐Abina, S., & Cavazzana, M. (2010) · Nature Immunology · doi:10.1038/ni0610-457 · Cited by 167

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

Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence

Cavalieri, S., Cazzaniga, S., Geuna, M., Magnani, Z. I., Bordignon, C., Naldini, L., & Bonini, C. (2003) · Blood · doi:10.1182/blood-2003-01-0297 · Cited by 157

IL-7 and IL-15 allow the generation of suicide gene–modified alloreactive self-renewing central memory human T lymphocytes

Kaneko, S., Mastaglio, S., Bondanza, A., Ponzoni, M., Sanvito, F., Aldrighetti, L. A., Radrizzani, M., Catamancio, S. L. S., Provasi, E., Mondino, A., et al. (2008) · Blood · doi:10.1182/blood-2008-05-156059 · Cited by 156

Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells

Calmels, B., Ferguson, C., Laukkanen, M. O., Adler, R. L., Faulhaber, M., Kim, H.-J., Sellers, S. E., Hematti, P., Schmidt, M., Kalle, C. V., et al. (2005) · Blood · doi:10.1182/blood-2005-03-1115 · Cited by 148

Analysis of Lentiviral Vector Integration in HIV+ Study Subjects Receiving Autologous Infusions of Gene Modified CD4+ T Cells

Wang, G. P., Levine, B. L., Binder, G. K., Berry, C. C., Malani, N., McGarrity, G., Tebas, P., June, C. H., & Bushman, F. D. (2009) · Molecular Therapy · doi:10.1038/mt.2009.16 · Cited by 147

Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment

Mazurier, F., Gan, O. I., McKenzie, J. L., Doedens, M., & Dick, J. E. (2003) · Blood · doi:10.1182/blood-2003-05-1558 · Cited by 146

Communication via gap junctions underlies early functional and beneficial interactions between grafted neural stem cells and the host

Jäderstad, J., Jäderstad, L. M., Li, J., Chintawar, S., Saltó, C., Pandolfo, M., Ourednik, V., Teng, Y. D., Sidman, R. L., Arenas, E., et al. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0915134107 · Cited by 141

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

Wynn, R., Wraith, J. E., Mercer, J., O’Meara, A., Tylee, K. L., Thornley, M., Church, H. J., & Bigger, B. (2009) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2008.11.005 · Cited by 134

Neuroprotection of Host Cells by Human Central Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis

Tamaki, S. J., Jacobs, Y., Dohse, M., Capela, A., Cooper, J. D., Reitsma, M. J., He, D., Tushinski, R. J., Belichenko, P. V., Salehi, A., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.022 · Cited by 134

The expanding universe of transposon technologies for gene and cell engineering

Ivics, Z., & Izsvák, Z. (2010) · Mobile DNA · doi:10.1186/1759-8753-1-25 · Cited by 134

Immunologic potential of donor lymphocytes expressing a suicide gene for early immune reconstitution after hematopoietic T-cell–depleted stem cell transplantation

Marktel, S., Magnani, Z. I., Ciceri, F., Cazzaniga, S., Riddell, S. R., Traversari, C., Bordignon, C., & Bonini, C. (2003) · Blood · doi:10.1182/blood-2002-08-2351 · Cited by 129

The φC31 Integrase System for Gene Therapy

Calos, M. P. (2006) · Current Gene Therapy · doi:10.2174/156652306779010642 · Cited by 128

High-throughput insertional mutagenesis screens in mice to identify oncogenic networks

Kool, J., & Berns, A. J. M. (2009) · Nature reviews. Cancer · doi:10.1038/nrc2647 · Cited by 128

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Wang, G. P., Berry, C. C., Malani, N., Leboulch, P., Fischer, A., Hacein‐Bey‐Abina, S., Cavazzana, M., & Bushman, F. D. (2010) · Blood · doi:10.1182/blood-2009-12-257352 · Cited by 125

Retrovirus-mediated gene transfer in primary T lymphocytes impairs their anti–Epstein-Barr virus potential through both culture-dependent and selection process–dependent mechanisms

Sauce, D., Bodinier, M., Garin, M. I., Petracca, B., Tonnelier, N., Duperrier, A., Melo, J. V., Apperley, J. F., Ferrand, C., Hervé, P., et al. (2002) · Blood · doi:10.1182/blood.v99.4.1165 · Cited by 122

Thrombopoietin, flt3, and kit ligands together suppress apoptosis of human mobilized CD34+ cells and recruit primitive CD34+Thy-1+ cells into rapid division

Murray, L. J., Young, J. C., Osborne, L. J., Luens, K. M., Scollay, R. G., & Hill, B. L. (1999) · Experimental Hematology · doi:10.1016/s0301-472x(99)00031-4 · Cited by 120

Insertional mutagenesis in gene therapy and stem cell biology

Baum, C. (2007) · Current Opinion in Hematology · doi:10.1097/moh.0b013e3281900f01 · Cited by 118

Adoptive T cell therapy of cancer

Brenner, M. K., & Heslop, H. E. (2010) · Current Opinion in Immunology · doi:10.1016/j.coi.2010.01.020 · Cited by 117

Epithelial stem cells in corneal regeneration and epidermal gene therapy

Pellegrini, G., Rama, P., Mavilio, F., & Luca, M. D. (2008) · The Journal of Pathology · doi:10.1002/path.2441 · Cited by 116

Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates

Beard, B. C., Trobridge, G. D., Ironside, C. M., McCune, J. S., Adair, J. E., & Kiem, H. (2010) · Journal of Clinical Investigation · doi:10.1172/jci40767 · Cited by 114

Refined human artificial chromosome vectors for gene therapy and animal transgenesis

Kazuki, Y., Hoshiya, H., Takiguchi, M., Abe, S., Iida, Y., Osaki, M., Katoh, M., Hiratsuka, M., Shirayoshi, Y., Hiramatsu, K., et al. (2010) · Gene Therapy · doi:10.1038/gt.2010.147 · Cited by 112

Integration profile of retroviral vector in gene therapy treated patients is cell‐specific according to gene expression and chromatin conformation of target cell

Biasco, L., Ambrosi, A., Pellin, D., Bartholomae, C. C., Brigida, I., Roncarolo, M. G., Serio, C. D., Kalle, C. V., Schmidt, M., & Aiuti, A. (2010) · EMBO Molecular Medicine · doi:10.1002/emmm.201000108 · Cited by 104

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

Wang, G. P., Garrigue, A., Ciuffi, A., Ronen, K., Leipzig, J. N., Berry, C. C., Lagresle‐Peyrou, C., Benjelloun, F., Hacein‐Bey‐Abina, S., Fischer, A., et al. (2008) · Nucleic Acids Research · doi:10.1093/nar/gkn125 · Cited by 104

Restriction of retroviral replication by APOBEC3G/F and TRIM5α

Huthoff, H., & Towers, G. J. (2008) · Trends in Microbiology · doi:10.1016/j.tim.2008.08.013 · Cited by 98

T Cell Receptor Gene Therapy for Cancer

Schmitt, T. M., Ragnarsson, G. B., & Greenberg, P. D. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.146 · Cited by 98

Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16

Modlich, U., Schambach, A., Brugman, M. H., Wicke, D. C., Knoess, S., Li, Z., Maetzig, T., Rudolph, C., Schlegelberger, B., & Baum, C. (2008) · Leukemia · doi:10.1038/leu.2008.118 · Cited by 96

Molecular Evidence of Lentiviral Vector-Mediated Gene Transfer into Human Self-Renewing, Multi-potent, Long-Term NOD/SCID Repopulating Hematopoietic Cells

Ailles, L., Schmidt, M., de Sio, F. R. S., Glimm, H., Cavalieri, S., Bruno, S., Piacibello, W., Kalle, C. V., & Naldini, L. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0720 · Cited by 96

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors

Izsvák, Z. (2009) · Methods · doi:10.1016/j.ymeth.2009.07.001 · Cited by 95

Gene Therapy Fulfilling Its Promise

Kohn, D. B., & Candotti, F. (2009) · New England Journal of Medicine · doi:10.1056/nejme0809614 · Cited by 94

Adoptive Cell Therapy: Genetic Modification to Redirect Effector Cell Specificity

Morgan, R. A., Dudley, M. E., & Rosenberg, S. A. (2010) · The Cancer Journal · doi:10.1097/ppo.0b013e3181eb3879 · Cited by 94

Retroviral vector insertion sites associated with dominant hematopoietic clones mark “stemness” pathways

Kustikova, O. S., Geiger, H., Li, Z., Brugman, M. H., Chambers, S. M., Shaw, C. A., Pike‐Overzet, K., de Ridder, D., Staal, F. J. T., von Keudell, G., et al. (2006) · Blood · doi:10.1182/blood-2006-08-044156 · Cited by 92

Long-Term Clinical and Molecular Follow-up of Large Animals Receiving Retrovirally Transduced Stem and Progenitor Cells: No Progression to Clonal Hematopoiesis or Leukemia

Kiem, H., Sellers, S. E., Thomasson, B. M., Morris, J. C., Tisdale, J. F., Horn, P. A., Hematti, P., Adler, R. L., Kuramoto, K., Calmels, B., et al. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2003.12.006 · Cited by 92

In vivo selection of MGMT(P140K) lentivirus–transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning

Zielske, S. P., Reese, J., Lingas, K. T., Donze, J. R., & Gerson, S. L. (2003) · Journal of Clinical Investigation · doi:10.1172/jci17922 · Cited by 87

Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs

Sorrentino, B. P. (2002) · Nature reviews. Cancer · doi:10.1038/nrc823 · Cited by 82

Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region

Arumugam, P. I., Higashimoto, T., Urbinati, F., Modlich, U., Nestheide, S. V., Xia, P., Fox, C. F., Corsinotti, A., Baum, C., & Malik, P. (2009) · Molecular Therapy · doi:10.1038/mt.2009.183 · Cited by 80

Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction

de Sio, F. R. S., Cascio, P., Zingale, A., Gasparini, M., & Naldini, L. (2006) · Blood · doi:10.1182/blood-2005-10-4047 · Cited by 80

Bone marrow and circulating stem/progenitor cells for regenerative cardiovascular therapy

Alaiti, M. A., Ishikawa, M., & Costa, M. A. (2010) · Translational research · doi:10.1016/j.trsl.2010.06.008 · Cited by 70

Somatic mosaicism in primary immune deficiencies

Wada, T., & Candotti, F. (2008) · Current Opinion in Allergy and Clinical Immunology · doi:10.1097/aci.0b013e328314b651 · Cited by 67

Cell-intrinsic and Vector-related Properties Cooperate to Determine the Incidence and Consequences of Insertional Mutagenesis

Kustikova, O. S., Schiedlmeier, B., Brugman, M. H., Stahlhut, M., Bartels, S., Li, Z., & Baum, C. (2009) · Molecular Therapy · doi:10.1038/mt.2009.134 · Cited by 67

Engineering Antigen-Specific T Cells from Genetically Modified Human Hematopoietic Stem Cells in Immunodeficient Mice

Kitchen, S. G., Bennett, M. S., Galić, Z., Kim, J., Xu, Q., Young, A. J., Lieberman, A., Joseph, A., Goldstein, H., Ng, H. L., et al. (2009) · PLoS ONE · doi:10.1371/journal.pone.0008208 · Cited by 62

Platelets as delivery systems for disease treatments

Shi, Q., & Montgomery, R. R. (2010) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2010.06.007 · Cited by 60

Genetically Modified CD34+ Hematopoietic Stem Cells Contribute to Turnover of Brain Perivascular Macrophages in Long-Term Repopulated Primates

Soulas, C., Donahue, R. E., Dunbar, C. E., Persons, D. A., Alvarez, X. A., & Williams, K. C. (2009) · American Journal Of Pathology · doi:10.2353/ajpath.2009.081010 · Cited by 55

In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene

Larochelle, A., Choi, U., Shou, Y., Naumann, N., Loktionova, N. A., Clevenger, J. R., Krouse, A. E., Metzger, M. E., Donahue, R. E., Kang, E. M., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37506 · Cited by 55

Large animal models of hematopoietic stem cell gene therapy

Trobridge, G. D., & Kiem, H. (2010) · Gene Therapy · doi:10.1038/gt.2010.47 · Cited by 52

Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients

Jacome, A., Navarro, S., Río, P., Yañez, R. M., González‐Murillo, Á., Lozano, M. L., Lamana, M. L., Sevilla, J., Olivé, T., de Heredia, C. D., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.26 · Cited by 51

High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells After Donor Lymphocyte Infusion

Cattoglio, C., Maruggi, G., Bartholomae, C. C., Malani, N., Pellin, D., Cocchiarella, F., Magnani, Z. I., Ciceri, F., Ambrosi, A., Kalle, C. V., et al. (2010) · PLoS ONE · doi:10.1371/journal.pone.0015688 · Cited by 47

Cytokines, including stem cell factor alone, enhance lentiviral transduction in nondividing human LTCIC and NOD/SCID repopulating cells

Zielske, S. P., & Gerson, S. L. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00005-4 · Cited by 41

Bone Marrow Transplantation for Primary Immunodeficiency Diseases

Szabolcs, P., Cavazzana, M., Fischer, A., & Veys, P. A. (2010) · Pediatric Clinics of North America · doi:10.1016/j.pcl.2009.12.004 · Cited by 41

Real-Time Definition of Non-Randomness in the Distribution of Genomic Events

Abel, U. R., Deichmann, A., Bartholomae, C. C., Schwarzwaelder, K., Glimm, H., Howe, S. J., Thrasher, A. J., Garrigue, A., Hacein‐Bey‐Abina, S., Cavazzana, M., et al. (2007) · PLoS ONE · doi:10.1371/journal.pone.0000570 · Cited by 31

Importance of Murine Study Design for Testing Toxicity of Retroviral Vectors in Support of Phase I Trials

Will, E., Bailey, J., Schuesler, T., Modlich, U., Balcik, B., Burzynski, B. B., Witte, D. P., Layh‐Schmitt, G., Rudolph, C., Schlegelberger, B., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300083 · Cited by 25

Transplantation of mouse HSCs genetically modified to express a CD4-restricted TCR results in long-term immunity that destroys tumors and initiates spontaneous autoimmunity

Ha, S. P., Klemen, N. D., Kinnebrew, G. H., Brandmaier, A., Marsh, J. W., Hangoc, G., Palmer, D. C., Restifo, N. P., Cornetta, K. G., Broxmeyer, H. E., et al. (2010) · Journal of Clinical Investigation · doi:10.1172/jci43274 · Cited by 22

NOD/SCID repopulating cells contribute only to short-term repopulation in the baboon

Mezquita, P., Beard, B., & Kiem, H. (2008) · Gene Therapy · doi:10.1038/gt.2008.108 · Cited by 19

Assays to Evaluate the Genotoxicity of Retroviral Vectors

Nienhuis, A. W. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.08.003 · Cited by 19

Site-specific integration of retroviral DNA in human cells using fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc-finger protein E2C

Su, K., Wang, D., Ye, J., Kim, Y. C., & Chow, S. A. (2009) · Methods · doi:10.1016/j.ymeth.2009.01.001 · Cited by 17

Stable Marking and Transgene Expression Without Progression to Monoclonality in Canine Long-Term Hematopoietic Repopulating Cells Transduced with Lentiviral Vectors

Enssle, J., Trobridge, G. D., Keyser, K. A., Ironside, C. M., Beard, B. C., & Kiem, H. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.076 · Cited by 13

Repetitive Busulfan Administration After Hematopoietic Stem Cell Gene Therapy Associated with a Dominant HDAC7 Clone in a Nonhuman Primate

Xie, J., Larochelle, A., Marić, I., Faulhaber, M., Donahue, R. E., & Dunbar, C. E. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.191 · Cited by 6

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science · doi:10.1126/science.1233158 · Cited by 1,180

Gene therapy returns to centre stage

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

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

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

Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges

Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893

In vitro and ex vivo strategies for intracellular delivery

Stewart, M. P., Sharei, A., Ding, X., Sahay, G., Langer, R., & Jensen, K. F. (2016) · Nature · doi:10.1038/nature19764 · Cited by 885