Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

<i>LMO2</i> -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 · see citing works

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

MORITA, S., Kojima, T., & Kitamura, T. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301206 · Cited by 1,660 · see citing works

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 1,189 · see citing works

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

Bell, A. C., West, A. G., & Felsenfeld, G. (1999) · Cell · doi:10.1016/s0092-8674(00)81967-4 · Cited by 1,101 · see citing works

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 699 · see citing works

Murine Leukemia Induced by Retroviral Gene Marking

Li, Z., Düllmann, J., Schiedlmeier, B., Schmidt, M., Kalle, C. V., Meyer, J., Forster, M., Stocking, C., Wahlers, A., Frank, O., et al. (2002) · Science · doi:10.1126/science.1068893 · Cited by 653 · see citing works

The Basic Science of Oncology

Tannock, I. F., Hill, R. P., & Carey, R. W. (1989) · Plastic & Reconstructive Surgery · doi:10.1097/00006534-198905000-00037 · Cited by 473 · see citing works

Genotoxicity of retroviral integration in hematopoietic cells.

Nienhuis, A., Dunbar, C., & Sorrentino, B. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.001 · Cited by 341 · see citing works

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 · see citing works

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 · see citing works

Use of the human elongation factor 1α promoter as a versatile and efficient expression system

Kim, D. W., Uetsuki, T., Kaziro, Y., Yamaguchi, N., & Sugano, S. (1990) · Gene · doi:10.1016/0378-1119(90)90091-5 · Cited by 277 · see citing works

Hot spots of retroviral integration in human CD34+ hematopoietic cells

Cattoglio, C., Facchini, G., Sartori, D., Antonelli, A., Miccio, A., Cassani, B., Schmidt, M., Kalle, C. V., Howe, S., Thrasher, A. J., et al. (2007) · Blood · doi:10.1182/blood-2007-01-068759 · Cited by 271 · see citing works

A chromatin insulator protects retrovirus vectors from chromosomal position effects

Emery, D. W., Yannaki, E., Tubb, J., & Stamatoyannopoulos, G. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.160159597 · Cited by 242 · see citing works

High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors

Salmon, P., Kindler, V., Ducrey, O., Chapuis, B., Zubler, R. H., & Trono, D. (2000) · Blood · doi:10.1182/blood.v96.10.3392 · Cited by 232 · see citing works

Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine–DNA methyltransferase in hematopoietic cells

Schambach, A., Bohne, J., Chandra, S., Will, E., Margison, G. P., Williams, D. A., & Baum, C. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.012 · Cited by 197 · see citing works

Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells

Du, Y., Jenkins, N. A., & Copeland, N. G. (2005) · Blood · doi:10.1182/blood-2005-03-1113 · Cited by 188 · see citing works

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 185 · see citing works

The Dark Side of EGFP: Defective Polyubiquitination

Baens, M., Noels, H., Broeckx, V., Hagens, S., Fevery, S., Billiau, A. D., Vankelecom, H., & Marynen, P. (2006) · PLoS ONE · doi:10.1371/journal.pone.0000054 · Cited by 149 · see citing works

Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression

Schambach, A., Bohne, J., Baum, C., Hermann, F. G., Egerer, L., von Laer, D., & Giroglou, T. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302698 · Cited by 145 · see citing works

Cooperating cancer-gene identification through oncogenic-retrovirus-induced insertional mutagenesis.

Du, Y., Spence, S., Jenkins, N., & Copeland, N. (2005) · Blood · doi:10.1182/blood-2004-12-4840 · Cited by 145 · see citing works

Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors

Schambach, A., Mueller, D., Galla, M., Verstegen, M. M. A., Wagemaker, G., Loew, R., Baum, C., & Bohne, J. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302807 · Cited by 143 · see citing works

Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque

Seggewiss, R. (2006) · Blood · doi:10.1182/blood-2005-10-4108 · Cited by 131 · see citing works

Improving Transcriptional Termination of Self-inactivating Gamma-retroviral and Lentiviral Vectors

Schambach, A., Galla, M., Maetzig, T., Loew, R., & Baum, C. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300152 · Cited by 127 · see citing works

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy.

Shou, Y., Ma, Z.-J., Lu, T., & Sorrentino, B. (2006) · Proceedings of the National Academy of Sciences of the United States of America · doi:10.1073/pnas.0603635103 · Cited by 116 · see citing works

Extended Core Sequences from the cHS4 Insulator Are Necessary for Protecting Retroviral Vectors from Silencing Position Effects

Aker, M., Tubb, J., Groth, A. C., Bukovsky, A. A., Bell, A. C., Felsenfeld, G., Kiem, H., Stamatoyannopoulos, G., & Emery, D. W. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.021 · Cited by 92 · see citing works

Characterization of the regulatory elements in the promoter of the human elongation factor-1 alpha gene.

Wakabayashi-Ito, N., & Nagata, S. (1994) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)43956-7 · Cited by 86 · see citing works

Suppression of Clonal Dominance in Cultured Human Lymphoid Cells by Addition of the cHS4 Insulator to a Lentiviral Vector

Evans‐Galea, M. V., Wielgosz, M. M., Hanawa, H., Srivastava, D. K., & Nienhuis, A. W. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300103 · Cited by 75 · see citing works

Predictable and efficient retroviral gene transfer into murine bone marrow repopulating cells using a defined vector dose.

Li, Z.-X., Schwieger, M., Lange, C., Kraunus, J., Sun, H.-Y., van den Akker, E. V. D., Modlich, U., Serinsöz, E., Will, E., von Laer, D., et al. (2003) · Experimental Hematology · doi:10.1016/j.exphem.2003.08.008 · Cited by 65 · see citing works

The potent enhancer activity of the polycythemic strain of spleen focus-forming virus in hematopoietic cells is governed by a binding site for Sp1 in the upstream control region and by a unique enhancer core motif, creating an exclusive target for PEBP/CBF

Baum, C., Itoh, K., Meyer, J., Laker, C., Ito, Y., & Ostertag, W. (1997) · Journal of Virology · doi:10.1128/jvi.71.9.6323-6331.1997 · Cited by 49 · see citing works

Promoter Choice for Retroviral Vectors: Transcriptional Strength Versus Trans-Activation Potential

Weber, E. L., & Cannon, P. M. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.067 · Cited by 32 · see citing works

Murine Retroviral but not Human Cellular Promoters Induce In Vivo Erythroid-specific Deregulation that can be Partially Prevented by Insulators

Richard, É., Richard, E., Malik, P., Ged, C., de Verneuil, H., & Moreau‐Gaudry, F. (2006) · Molecular Therapy · doi:10.1038/sj.mt.6300030 · Cited by 30 · see citing works

Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways.

Kustikova, O., Geiger, H., Li, Z.-X., Brugman, M., Chambers, S. M., Shaw, C. A., Pike-Overzet, K., de Ridder, D., Staal, F., von Keudell, G., et al. (2007) · Blood · doi:10.1182/blood-2006-08-044156 · Cited by 22 · see citing works

In Vivo Analysis of Retroviral Enhancer Mutations in Hematopoietic Cells: SP1/EGR1 and ETS/GATA Motifs Contribute to Long Terminal Repeat Specificity

Wahlers, A., Zipfel, P. F., Schwieger, M., Ostertag, W., & Baum, C. (2002) · Journal of Virology · doi:10.1128/jvi.76.1.303-312.2002 · Cited by 16 · see citing works

Upstream Conserved Sequences of Mouse Leukemia Viruses Are Important for High Transgene Expression in Lymphoid and Hematopoietic Cells

Wahlers, A., Kustikova, O. S., Zipfel, P. F., Itoh, K., Koester, M., Heberlein, C., Li, Z., Schiedlmeier, B., Skerka, C., Fehse, B., et al. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0671 · Cited by 15 · see citing works

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 · see citing works

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 · see citing works

Gene therapy returns to centre stage

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

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

Ex vivo gene transfer and correction for cell-based therapies

Naldini, L. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2985 · Cited by 386 · see citing works

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 348 · see citing works

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 282 · see citing works

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 · see citing works

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 · see citing works

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 213 · see citing works

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 · see citing works

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 186 · see citing works

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 178 · see citing works

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 · see citing works

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β ^A(T87Q) - <i>Globin</i> 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 · see citing works

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 155 · see citing works

Gammaretroviral Vectors: Biology, Technology and Application

Maetzig, T., Galla, M., Baum, C., & Schambach, A. (2011) · Viruses · doi:10.3390/v3060677 · Cited by 166 · see citing works

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 149 · see citing works

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 135 · see citing works

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 · see citing works

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. E., Verheust, C., Debyser, Z., & Herman, P. (2009) · Current Gene Therapy · doi:10.2174/156652309790031120 · Cited by 126 · see citing works

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 124 · see citing works

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 120 · see citing works

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 118 · see citing works

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 · see citing works

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 · see citing works

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

Irving, M., Lanitis, E., Migliorini, D., Ivics, Z., & Guedan, S. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.173 · Cited by 86 · see citing works

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 109 · see citing works

SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa.

Titeux, M., Pendaries, V., Zanta-Boussif, M., Décha, A., Pironon, N., Tonasso, L., Mejía, J., Brice, A., Danos, O., & Hovnanian, A. (2010) · Molecular Therapy · doi:10.1038/mt.2010.91 · Cited by 118 · see citing works

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

Cavazzana, M., Six, E. M., Lagresle‐Peyrou, C., André‐Schmutz, I., & Hacein‐Bey‐Abina, S. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.137 · Cited by 104 · see citing works

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 101 · see citing works

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 · see citing works

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 · see citing works

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 102 · see citing works

Gene therapy of inherited diseases

Fischer, A., & Cavazzana, M. (2008) · The Lancet · doi:10.1016/s0140-6736(08)60874-0 · Cited by 96 · see citing works

Gene Therapy Fulfilling Its Promise

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

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 92 · see citing works

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 91 · see citing works

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 89 · see citing works

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

Pike-Overzet, K., Rodijk, M., Ng, Y.-Y., Baert, M., Lagresle-Peyrou, C., Schambach, A., Zhang, F., Hoeben, R., Hacein-Bey-Abina, S., Lankester, A., et al. (2011) · Leukemia · doi:10.1038/leu.2011.106 · Cited by 83 · see citing works

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 91 · see citing works

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 86 · see citing works

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 85 · see citing works

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 85 · see citing works

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 · see citing works

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 79 · see citing works

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 · see citing works

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 79 · see citing works

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 75 · see citing works

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 78 · see citing works

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

Arumugam, P., Higashimoto, T., Urbinati, F., Modlich, U., Nestheide, S., Xia, P., Fox, C. F., Corsinotti, A., Baum, C., & Malik, P. (2009) · Molecular Therapy · doi:10.1038/mt.2009.183 · Cited by 15 · see citing works

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 73 · see citing works

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 71 · see citing works

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 70 · see citing works

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 · see citing works

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 · see citing works

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 67 · see citing works

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 67 · see citing works

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 66 · see citing works

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 · see citing works

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 · see citing works

Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis.

Kustikova, O., Schiedlmeier, B., Brugman, M., Stahlhut, M., Bartels, S., Li, Z.-X., & Baum, C. (2009) · Molecular Therapy · doi:10.1038/mt.2009.134 · Cited by 72 · see citing works

Update on Gene Therapy for Immunodeficiencies

Kohn, D. (2010) · Clinical Immunology · doi:10.1016/j.clim.2009.12.003 · Cited by 66 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 62 · see citing works

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 64 · see citing works

Design and potential of non-integrating lentiviral vectors

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

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

Al-Allaf, F., Coutelle, C., Waddington, S., David, A., Harbottle, R., & Themis, M. (2010) · International Archives of Medicine · doi:10.1186/1755-7682-3-36 · Cited by 63 · see citing works

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 · see citing works

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., Baum, C., et al. (2009) · Gene Therapy · doi:10.1038/gt.2008.143 · Cited by 63 · see citing works

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 59 · see citing works

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 58 · see citing works

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., & Grez, M. (2011) · Gene Therapy · doi:10.1038/gt.2011.167 · Cited by 60 · see citing works

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., Donahue, R., & Tisdale, J. (2009) · Journal of Virology · doi:10.1128/jvi.00357-09 · Cited by 57 · see citing works

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 55 · see citing works

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 · see citing works

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 50 · see citing works

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., Hauser, H., Stein, S., Grez, M., Thornhill, S., Thrasher, A., et al. (2010) · Gene Therapy · doi:10.1038/gt.2009.134 · Cited by 59 · see citing works

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 49 · see citing works

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., Gueven, S., Centola, M., Tsaryk, R., Barbeck, M., Stuedle, C., Barbero, A., Helmrich, U., et al. (2016) · Stem Cells Translational Medicine · doi:10.5966/sctm.2015-0321 · Cited by 51 · see citing works

Retroviral Vectors for Gene Therapy

Maier, P., Kalle, C. V., & Laufs, S. (2010) · Future Microbiology · doi:10.2217/fmb.10.100 · Cited by 49 · see citing works

Lentiviral Vector Gene Therapy: Effective and Safe?

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

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 46 · see citing works

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 47 · see citing works

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 · see citing works

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 44 · see citing works

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 · see citing works

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 · see citing works

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 43 · see citing works

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 · see citing works

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 43 · see citing works

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 · see citing works

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 40 · see citing works

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 40 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 79 · see citing works

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 · see citing works