Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

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

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

Plat-E: an efficient and stable system for transient packaging of retroviruses

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

Ott, M., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A. J., Kunkel, H., et al. (2006) · Nature Medicine · doi:10.1038/nm1393 · Cited by 1,202

The protein CTCF is required for the enhancer blocking activity of vertebrate insulators

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

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Murine Leukemia Induced by Retroviral Gene Marking

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The Basic Science of Oncology

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

Modlich, U., Bohne, J., Schmidt, M., Kalle, C. V., Knöß, S., Schambach, A., & Baum, C. (2006) · Blood · doi:10.1182/blood-2005-08-024976 · Cited by 326

Clonal Dominance of Hematopoietic Stem Cells Triggered by Retroviral Gene Marking

Kustikova, O. S., Fehse, B., Modlich, U., Yang, M., Düllmann, J., Kamino, K., Neuhoff, N. V., Schlegelberger, B., Li, Z., & Baum, C. (2005) · Science · doi:10.1126/science.1105063 · Cited by 323

Genotoxicity of Retroviral Integration In Hematopoietic Cells

Nienhuis, A. W., Dunbar, C. E., & Sorrentino, B. P. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.001 · Cited by 301

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

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

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

Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

Heckl, D., Kowalczyk, M. S., Yudovich, D., Belizaire, R., Puram, R. V., McConkey, M. E., Thielke, A., Aster, J. C., Regev, A., & Ebert, B. L. (2014) · Nature Biotechnology · doi:10.1038/nbt.2951 · Cited by 555

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1404588 · Cited by 412

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

Ex vivo gene transfer and correction for cell-based therapies

Naldini, L. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2985 · Cited by 395

Wiskott‐Aldrich syndrome: a comprehensive review

Massaad, M. J., Ramesh, N., & Geha, R. S. (2013) · Annals of the New York Academy of Sciences · doi:10.1111/nyas.12049 · Cited by 358

Gene therapy on the move

Kaufmann, K. B., Büning, H., Galy, A. H. M., Schambach, A., & Grez, M. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201202287 · Cited by 300

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Kohn, D. B., Booth, C., Shaw, K. L., Xu‐Bayford, J., Garabedian, E., Trevisan, V., Carbonaro-Sarracino, D. A., Soni, K., Terrazas, D., Snell, K., et al. (2021) · New England Journal of Medicine · doi:10.1056/nejmoa2027675 · Cited by 223

Integration-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

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

Sabatino, D. E., Bushman, F. D., Chandler, R. J., Crystal, R. G., Davidson, B. L., Dolmetsch, R. E., Eggan, K. C., Gao, G., Gil-Fariña, I., Kay, M. A., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.06.004 · Cited by 222

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.3 · Cited by 189

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical Ex Vivo Gene Therapy Application

Merten, O.-W., Charrier, S., Laroudie, N., Fauchille, S., Dugué, C., Jenny, C., Audit, M., Zanta, M.-A., Chautard, H., Radrizzani, M., et al. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.060 · Cited by 191

Limitations in the Design of Chimeric Antigen Receptors for Cancer Therapy

Stoiber, S., Cadilha, B. L., Benmebarek, M.-R., Lesch, S., Endres, S., & Kobold, S. (2019) · Cells · doi:10.3390/cells8050472 · Cited by 182

Biosafety Features of Lentiviral Vectors

Schambach, A., Zychlinski, D., Ehrnstroem, B., & Baum, C. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.229 · Cited by 173

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β A(T87Q) - Globin Gene

Negre, O., Eggimann, A.-V., Beuzard, Y., Ribeil, J.-A., Bourget, P., Borwornpinyo, S., Hongeng, S., Hacein-Bey, S., Cavazzana, M., Leboulch, P., et al. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.007 · Cited by 159

Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives

Vargas, J. E., Chicaybam, L., Stein, R. T., Tanuri, A., Cañedo, A. D., & Bonamino, M. (2016) · Journal of Translational Medicine · doi:10.1186/s12967-016-1047-x · Cited by 165

Gammaretroviral Vectors: Biology, Technology and Application

Maetzig, T., Galla, M., Baum, C., & Schambach, A. (2011) · Viruses · doi:10.3390/v3060677 · Cited by 161

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

Throm, R. E., Ouma, A. A., Zhou, S., Chandrasekaran, A., Lockey, T. D., Greene, M. R., Ravin, S. S. D., Moayeri, M., Malech, H. L., Sorrentino, B. P., et al. (2009) · Blood · doi:10.1182/blood-2008-11-191049 · Cited by 154

Retroviral and Lentiviral Safety Analysis of Gene-Modified T Cell Products and Infused HIV and Oncology Patients

Marcucci, K. T., Jadlowsky, J. K., Hwang, W., Suhoski, M. M., Gonzalez, V. E., Kulikovskaya, I. M., Gupta, M., Lacey, S. F., Plesa, G., Chew, A., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.10.012 · Cited by 137

Brief Reports: Lysosomal Cross-Correction by Hematopoietic Stem Cell-Derived Macrophages Via Tunneling Nanotubes

Naphade, S. B., Sharma, J., Chevronnay, H. P. G., Shook, M. A., Yeagy, B. A., Rocca, C. J., Ur, S. N., Lau, A., Courtoy, P. J., & Cherqui, S. (2014) · Stem Cells · doi:10.1002/stem.1835 · Cited by 131

State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations

Pauwels, K., Gijsbers, R., Toelen, J., Schambach, A., Willard-Gallo, K., Verheust, C., Debyser, Z., & Herman, P. (2009) · Current Gene Therapy · doi:10.2174/156652309790031120 · Cited by 106

INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes

Sherman, E. A., Nobles, C. L., Berry, C. C., Six, E. M., Wu, Y., Dryga, A., Malani, N., Male, F., Reddy, S. G., Bailey, A., et al. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2016.11.002 · Cited by 128

miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

Guda, S., Brendel, C., Renella, R., Du, P., Bauer, D. E., Canver, M. C., Grenier, J. K., Grimson, A., Kamran, S. C., Thornton, J. E., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.113 · Cited by 124

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

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

Zhou, S., Mody, D., DeRavin, S. S., Hauer, J., Lu, T., Ma, Z., Hacein-Bey Abina, S., Gray, J. T., Greene, M. R., Cavazzana-Calvo, M., et al. (2010) · Blood · doi:10.1182/blood-2009-10-250209 · Cited by 119

Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

Wu, C., & Dunbar, C. E. (2011) · Frontiers of Medicine · doi:10.1007/s11684-011-0159-1 · Cited by 114

Choosing the Right Tool for Genetic Engineering: Clinical Lessons from Chimeric Antigen Receptor-T Cells

Irving, M. B., Lanitis, E., Migliorini, D., Ivics, Z., & Guedan, S. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.173 · Cited by 112

Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

Piguet, F., Alves, S., & Cartier, N. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.160 · Cited by 115

SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa

Titeux, M., Pendaries, V., Zanta, M. A., Décha, A., Pironon, N., Tonasso‐Calvière, L., Mejía, J. E., Brice, A., Danos, O., & Hovnanian, A. (2010) · Molecular Therapy · doi:10.1038/mt.2010.91 · Cited by 114

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

Cavazzana, M., Six, E., Lagresle-Peyrou, C., André-Schmutz, I., & Hacein-Bey-Abina, S. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.137 · Cited by 106

Current and future alternative therapies for beta-thalassemia major

Dreuzy, E. D., Bhukhai, K., Leboulch, P., & Payen, E. (2016) · Biomedical Journal · doi:10.1016/j.bj.2015.10.001 · Cited by 104

Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

Wilber, A. C., Hargrove, P. W., Kim, Y., Riberdy, J. M., Sankaran, V. G., Papanikolaou, E., Georgomanoli, M., Anagnou, N. P., Orkin, S. H., Nienhuis, A. W., et al. (2010) · Blood · doi:10.1182/blood-2010-08-300723 · 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 98

Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations

Hołubowicz, R., Du, S. W., Felgner, J. H., Šmidák, R., Choi, E. H., Palczewska, G., Menezes, C. R., Dong, Z., Gao, F., Medani, O., et al. (2024) · Nature Biomedical Engineering · doi:10.1038/s41551-024-01296-2 · Cited by 111

Gene therapy of inherited diseases

Fischer, A., & Cavazzana, M. (2008) · The Lancet · doi:10.1016/s0140-6736(08)60874-0 · Cited by 98

Gene Therapy Fulfilling Its Promise

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

The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors

Emery, D. W. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.233 · Cited by 98

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

van Til, N. P., Stok, M., Aerts‐Kaya, F., de Waard, M. C., Farahbakhshian, E., Visser, T. P., Kroos, M. A., Jacobs, E. H., Willart, M. A. M., van der Wegen, P., et al. (2010) · Blood · doi:10.1182/blood-2009-11-252874 · Cited by 94

PD-1+ natural killer cells in human non-small cell lung cancer can be activated by PD-1/PD-L1 blockade

Trefny, M. P., Kaiser, M., Stanczak, M. A., Herzig, P., Savic, S., Wiese, M. N., Lardinois, D., Laubli, H. P., Uhlenbrock, F., & Zippelius, A. (2020) · Cancer Immunology Immunotherapy · doi:10.1007/s00262-020-02558-z · Cited by 88

Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

Pike‐Overzet, K., Rodijk, M., Ng, Y.-Y., Baert, M. R. M., Lagresle‐Peyrou, C., Schambach, A., Zhang, F., Hoeben, R. C., Hacein‐Bey‐Abina, S., Lankester, A. C., et al. (2011) · Leukemia · doi:10.1038/leu.2011.106 · Cited by 90

Strategies for Vaccination: Conventional Vaccine Approaches Versus New-Generation Strategies in Combination with Adjuvants

Bouazzaoui, A., Abdellatif, A. A. H., Al-Allaf, F. A., Bogari, N. M., Al-Dehlawi, S., & Qari, S. H. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13020140 · Cited by 92

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

Zanta, M.-A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A. J., Hope, T. J., & Galy, A. H. M. (2009) · Gene Therapy · doi:10.1038/gt.2009.3 · Cited by 89

Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

Carbonaro, D. A., Zhang, L., Jin, X., Montiel-Equihua, C., Geiger, S., Carmo, M., Cooper, A. R., Fairbanks, L. D., Kaufman, M. L., Sebire, N. J., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.265 · Cited by 86

Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis

Harrison, F., Yeagy, B. A., Rocca, C. J., Kohn, D. B., Salomon, D. R., & Cherqui, S. (2012) · Molecular Therapy · doi:10.1038/mt.2012.214 · Cited by 90

Primary Immune Deficiency Treatment Consortium (PIDTC) report

Griffith, L. M., Cowan, M. J., Notarangelo, L. D., Kohn, D. B., Puck, J. M., Pai, S., Ballard, B., Bauer, S. C., Bleesing, J., Boyle, M., et al. (2013) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2013.07.052 · Cited by 83

Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design

Maruggi, G., Porcellini, S., Facchini, G., Perna, S. K., Cattoglio, C., Sartori, D., Ambrosi, A., Schambach, A., Baum, C., Bonini, C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.51 · Cited by 81

Cocal-pseudotyped Lentiviral Vectors Resist Inactivation by Human Serum and Efficiently Transduce Primate Hematopoietic Repopulating Cells

Trobridge, G. D., Wu, R. A., Hansen, M. J., Ironside, C. M., Watts, K. L., Olsen, P., Beard, B. C., & Kiem, H. (2009) · Molecular Therapy · doi:10.1038/mt.2009.282 · Cited by 79

Clinical Applications of Gene Therapy for Primary Immunodeficiencies

Cicalese, M. P., & Aiuti, A. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.047 · Cited by 80

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich’s ataxia

Rocca, C. J., Goodman, S. M., Dulin, J. N., Haquang, J. H., Gertsman, I., Blondelle, J., Smith, J. L. M., Heyser, C. J., & Cherqui, S. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aaj2347 · Cited by 78

The effect of controlled expression of VEGF by transduced myoblasts in a cardiac patch on vascularization in a mouse model of myocardial infarction

Marsano, A., Maidhof, R., Luo, J., Fujikara, K., Konofagou, E. E., Banfi, A., & Vunjak‐Novakovic, G. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.09.038 · Cited by 79

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

Alpharetroviral Self-inactivating Vectors: Long-term Transgene Expression in Murine Hematopoietic Cells and Low Genotoxicity

Suerth, J. D., Maetzig, T., Brugman, M. H., Heinz, N., Appelt, J.-U., Kaufmann, K. B., Schmidt, M., Grez, M., Modlich, U., Baum, C., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.309 · Cited by 74

Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design

Suerth, J. D., Maetzig, T., Galla, M., Baum, C., & Schambach, A. (2010) · Journal of Virology · doi:10.1128/jvi.00182-10 · Cited by 74

Progress and prospects: gene therapy for inherited immunodeficiencies

Qasim, W., Gaspar, H. B., & Thrasher, A. J. (2009) · Gene Therapy · doi:10.1038/gt.2009.127 · Cited by 71

Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through

Gurumoorthy, N., Nordin, F., Tye, G. J., Wan Kamarul Zaman, W. S., & Ng, M. H. (2022) · Biomedicines · doi:10.3390/biomedicines10010107 · Cited by 69

Omega-1 knockdown in Schistosoma mansoni eggs by lentivirus transduction reduces granuloma size in vivo

Hagen, J., Young, N. D., Every, A. L., Pagel, C. N., Schnoeller, C., Scheerlinck, J. Y., Gasser, R. B., & Kalinna, B. H. (2014) · Nature Communications · doi:10.1038/ncomms6375 · Cited by 68

Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy

Díez, I. A., Zychlinski, D., Coci, E. G., Galla, M., Modlich, U., Dewey, R. A., Schwarzer, A., Maetzig, T., Mpofu, N., Jaeckel, E., et al. (2011) · Molecular Pharmaceutics · doi:10.1021/mp200132u · Cited by 69

Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

van Til, N. P., de Boer, H., Mashamba, N., Wabik, A., Huston, M. W., Visser, T. P., Fontana, E., Poliani, P. L., Cassani, B., Zhang, F., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.110 · Cited by 66

Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice

Dahl, M., Doyle, A., Olsson, K., Månsson, J., Marques, A. R. A., Mirzaian, M., Aerts, J. M. F. G., Ehinger, M., Rothe, M., Modlich, U., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.16 · Cited by 67

Combinatorial Incorporation of Enhancer-Blocking Components of the Chicken β-Globin 5′HS4 and Human T-Cell Receptor α/δ BEAD-1 Insulators in Self-Inactivating Retroviral Vectors Reduces Their Genotoxic Potential

Ramezani, A., Hawley, T. S., & Hawley, R. G. (2008) · Stem Cells · doi:10.1634/stemcells.2008-0258 · 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

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

Update on gene therapy for immunodeficiencies

Kohn, D. B. (2010) · Clinical Immunology · doi:10.1016/j.clim.2009.12.003 · Cited by 67

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy

Cassani, B., Montini, E., Maruggi, G., Ambrosi, A., Mirolo, M., Selleri, S., Biral, E., Frugnoli, I., Hernandez-Trujillo, V. P., Serio, C. D., et al. (2009) · Blood · doi:10.1182/blood-2009-02-202085 · Cited by 65

Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency

García-Gómez, M., Calabria, A., Garcı́a-Bravo, M., Benedicenti, F., Kosinski, P. A., López-Manzaneda, S., Hill, C., Mañú‐Pereira, M. D. M., Martín, M. A., Orman, I., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.87 · Cited by 64

The 3′ Region of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to Its Core and Is Required for Full Insulator Activity

Arumugam, P. I., Urbinati, F., Velu, C. S., Higashimoto, T., Grimes, H. L., & Malik, P. (2009) · PLoS ONE · doi:10.1371/journal.pone.0006995 · Cited by 62

Creating Higher Titer Lentivirus with Caffeine

Ellis, B. L., Potts, P. R., & Porteus, M. H. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.068 · Cited by 65

Engineering strategies to safely drive CAR T-cells into the future

Rossi, M., & Breman, E. (2024) · Frontiers in Immunology · doi:10.3389/fimmu.2024.1411393 · Cited by 68

Design and potential of non-integrating lentiviral vectors

Shaw, A., & Cornetta, K. (2014) · Biomedicines · doi:10.3390/biomedicines2010014 · Cited by 60

LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives

Al-Allaf, F. A., Coutelle, C., Waddington, S. N., David, A. L., Harbottle, R. P., & Themis, M. (2010) · International Archives of Medicine · doi:10.1186/1755-7682-3-36 · Cited by 60

Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease

Marquez Loza, L. I., Yuen, E. C., & McCray, P. B. (2019) · Genes · doi:10.3390/genes10030218 · Cited by 59

Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells

Moreno-Carranza, B., Gentsch, M., Stein, S., Schambach, A., Santilli, G., Rudolf, E., Ryser, M., Haria, S., Thrasher, A. J., Baum, C., et al. (2008) · Gene Therapy · doi:10.1038/gt.2008.143 · Cited by 61

Preventing and exploiting the oncogenic potential of integrating gene vectors

Modlich, U., & Baum, C. (2009) · Journal of Clinical Investigation · doi:10.1172/jci38831 · Cited by 61

Lymphomagenesis in SCID-X1 Mice Following Lentivirus-mediated Phenotype Correction Independent of Insertional Mutagenesis and γc Overexpression

Ginn, S. L., Liao, S. H. Y., Dane, A. P., Hu, M., Hyman, J., Finnie, J. W., Zheng, M., Cavazzana, M., Alexander, S. I., Thrasher, A. J., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.50 · Cited by 59

Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter

Brendel, C., Müller-Kuller, U., Schultze‐Strasser, S., Stein, S., Chen-Wichmann, L., Krattenmacher, A., Kunkel, H., Dillmann, A., Antoniou, M. N., & Grez, M. (2011) · Gene Therapy · doi:10.1038/gt.2011.167 · Cited by 62

Development of a Human Immunodeficiency Virus Type 1-Based Lentiviral Vector That Allows Efficient Transduction of both Human and Rhesus Blood Cells

Uchida, N., Washington, K. N., Hayakawa, J., Hsieh, M. M., Bonifacino, A. C., Krouse, A. E., Metzger, M. E., Donahue, R. E., & Tisdale, J. F. (2009) · Journal of Virology · doi:10.1128/jvi.00357-09 · Cited by 56

Polyclonal fluctuation of lentiviral vector–transduced and expanded murine hematopoietic stem cells

Maetzig, T., Brugman, M. H., Bartels, S., Heinz, N., Kustikova, O. S., Modlich, U., Li, Z., Galla, M., Schiedlmeier, B., Schambach, A., et al. (2011) · Blood · doi:10.1182/blood-2010-08-303222 · Cited by 56

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

Punwani, D., Kawahara, M., Yu, J., Sanford, U. R., Roy, S., Patel, K. P., Carbonaro, D. A., Karlen, A. D., Khan, S., Cornetta, K. G., et al. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.064 · Cited by 55

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

Poletti, V., Charrier, S., Corre, G., Gjata, B., Vignaud, A., Zhang, F., Rothe, M., Schambach, A., Gaspar, H. B., Thrasher, A. J., et al. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.03.002 · Cited by 51

A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating γ-retroviral vectors using targeted integration

Loew, R., Meyer, Y., Kuehlcke, K., Gama-Norton, L., Wirth, D., Häuser, H., Stein, S., Grez, M., Thornhill, S. I., Thrasher, A. J., et al. (2009) · Gene Therapy · doi:10.1038/gt.2009.134 · Cited by 51

Gene therapy for the Wiskott–Aldrich syndrome

Galy, A. H. M., & Thrasher, A. J. (2011) · Current Opinion in Allergy and Clinical Immunology · doi:10.1097/aci.0b013e32834c230c · Cited by 56

Spontaneous In Vivo Chondrogenesis of Bone Marrow-Derived Mesenchymal Progenitor Cells by Blocking Vascular Endothelial Growth Factor Signaling

Marsano, A., da Cunha, C. M. M., Ghanaati, S., Güven, S., Centola, M., Tsaryk, R., Barbeck, M., Stüdle, C., Barbero, A., Helmrich, U., et al. (2016) · Stem Cells Translational Medicine · doi:10.5966/sctm.2015-0321 · Cited by 50

Retroviral Vectors for Gene Therapy

Maier, P., Kalle, C. V., & Laufs, S. (2010) · Future Microbiology · doi:10.2217/fmb.10.100 · Cited by 51

Lentiviral Vector Gene Therapy: Effective and Safe?

Persons, D. A. (2010) · Molecular Therapy · doi:10.1038/mt.2010.70 · Cited by 49

BET-independent MLV-based Vectors Target Away From Promoters and Regulatory Elements

Ashkar, S. E., Rijck, J. D., Demeulemeester, J., Vets, S., Madlala, P., Čermáková, K., Debyser, Z., & Gijsbers, R. (2014) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2014.33 · Cited by 50

Transduction of Human CD34 + Repopulating Cells with a Self-Inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line

Greene, M. R., Lockey, T. D., Mehta, P. K., Kim, Y., Eldridge, P. W., Gray, J. T., & Sorrentino, B. P. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.150 · Cited by 50

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

Baruteau, J., Perocheau, D. P. B., Hanley, J., Lorvellec, M., Rocha‐Ferreira, E., Karda, R., Ng, J., Suff, N., Diaz, J. F. A., Rahim, A. A., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05972-1 · Cited by 45

The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

Piguet, F., Saint-Denis, T. D., Audouard, E., Beccaria, K., André, A., Wurtz, G., Schatz, R., Alves, S., Sevin, C., Zérah, M., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.105 · Cited by 47

Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease

Blanco, E., Izotova, N., Booth, C., & Thrasher, A. J. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.608653 · Cited by 45

Amelioration of murine β-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both γ-globin and the MGMT drug-resistance gene

Zhao, H., Pestina, T. I., Nasimuzzaman, M., Mehta, P. K., Hargrove, P. W., & Persons, D. A. (2009) · Blood · doi:10.1182/blood-2008-10-186684 · Cited by 43

Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque

Espinoza, D. A., Fan, X., Yang, D., Cordes, S., Truitt, L. L., Calvo, K. R., Yabe, I. M., Demirci, S., Hope, K. J., Hong, S. G., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.04.003 · Cited by 41

Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors

Maetzig, T., Galla, M., Brugman, M. H., Loew, R., Baum, C., & Schambach, A. (2009) · Gene Therapy · doi:10.1038/gt.2009.129 · Cited by 43

Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

Huston, M. W., van Til, N. P., Visser, T. P., Arshad, S., Brugman, M. H., Cattoglio, C., Nowrouzi, A., Li, Y., Schambach, A., Schmidt, M., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.127 · Cited by 41

Engineered Treg cells: The heir to the throne of immunotherapy

Stucchi, A., Maspes, F., Montee-Rodrigues, E., & Fousteri, G. (2023) · Journal of Autoimmunity · doi:10.1016/j.jaut.2022.102986 · Cited by 43

Optimization of Lentiviral Vectors Generation for Biomedical and Clinical Research Purposes: Contemporary Trends in Technology Development and Applications

Kumar, P., & Chan, W. (2011) · Current Gene Therapy · doi:10.2174/156652311794940782 · Cited by 42

Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy

Ago, Y., Rintz, E., Musini, K. S., Ma, Z., & Tomatsu, S. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25021113 · Cited by 44

Genetic modification of lymphocytes by retrovirus-based vectors

Suerth, J. D., Schambach, A., & Baum, C. (2012) · Current Opinion in Immunology · doi:10.1016/j.coi.2012.08.007 · Cited by 38

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

Modlich, U., Bohne, J., Schmidt, M., Kalle, C. V., Knöß, S., Schambach, A., & Baum, C. (2006) · Blood · doi:10.1182/blood-2005-08-024976 · Cited by 326

Promoters and insulators: creating a safer integrating vector

Weber, E. L. (2015) · University of Southern California Digital Library · doi:10.25549/usctheses-m587

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

Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design

Maruggi, G., Porcellini, S., Facchini, G., Perna, S. K., Cattoglio, C., Sartori, D., Ambrosi, A., Schambach, A., Baum, C., Bonini, C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.51 · Cited by 81

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.3 · Cited by 189