Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

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 · see citing works

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works

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 · see citing works

The Liver as an Immunological Organ

Racanelli, V., & Rehermann, B. (2006) · Hepatology · doi:10.1002/hep.21060 · Cited by 1,377 · see citing works

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 · 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

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 · see citing works

Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

Mingozzi, F., Liu, Y.-L., Dobrzynski, E., Kaufhold, A., Liu, J. H., Wang, Y., Arruda, V. R., High, K. A., & Herzog, R. W. (2003) · Journal of Clinical Investigation · doi:10.1172/jci16887 · Cited by 466 · see citing works

Effective gene therapy with nonintegrating lentiviral vectors

Yáñez-Muñoz, R. J., Balaggan, K. S., MacNeil, A., Howe, S. J., Schmidt, M., Smith, A. J., Buch, P., MacLaren, R. E., Anderson, P. N., Barker, S. E., et al. (2006) · Nature Medicine · doi:10.1038/nm1365 · Cited by 460 · see citing works

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)

Schmidt, M., Schwarzwaelder, K., Bartholomae, C., Zaoui, K., Ball, C., Pilz, I., Braun, S., Glimm, H., & von Kalle, C. (2007) · Nature Methods · doi:10.1038/nmeth1103 · Cited by 328 · see citing works

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

Palma, M. D., 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 · see citing works

Adeno-associated virus vectors integrate at chromosome breakage sites

Miller, D. G., Petek, L. M., & Russell, D. W. (2004) · Nature Genetics · doi:10.1038/ng1380 · Cited by 266 · see citing works

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

Brown, B. D., Cantore, A., Annoni, A., Sergi, L. S., Lombardo, A., Della Valle, P., D'Angelo, A., & Naldini, L. (2007) · Blood · doi:10.1182/blood-2007-03-078493 · Cited by 252 · see citing works

Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression <i>in vitro</i> and <i>in vivo</i>

Philippe, S., Sarkis, C., Barkats, M., Mammeri, H., Ladroue, C., Petit, C., Mallet, J., & Serguera, C. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0606197103 · Cited by 249 · see citing works

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

VandenDriessche, T., Thorrez, L., Naldini, L., Follenzi, A., Moons, L., Berneman, Z. N., Collen, D., & Chuah, M. (2002) · Blood · doi:10.1182/blood.v100.3.813 · Cited by 236 · see citing works

Human immunodeficiency virus type 1 integrase mutants retain in vitro integrase activity yet fail to integrate viral DNA efficiently during infection

Leavitt, A. D., Robles, G., Alesandro, N., & Varmus, H. E. (1996) · Journal of Virology · doi:10.1128/jvi.70.2.721-728.1996 · Cited by 232 · see citing works

Transient Gene Expression by Nonintegrating Lentiviral Vectors

Nightingale, S. J., Hollis, R. P., Pepper, K. A., Petersen, D., Yu, X.-J., Yang, C., Bahner, I., & Kohn, D. B. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.008 · Cited by 192 · see citing works

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 182 · see citing works

Stable Gene Transfer to Muscle Using Non-integrating Lentiviral Vectors

Apolonia, L., Waddington, S. N., Fernandes, C., Ward, N. J., Bouma, G., Blundell, M. P., Thrasher, A. J., Collins, M. K., & Philpott, N. J. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300281 · Cited by 174 · see citing works

Ectopic expression of neural autoantigen in mouse liver suppresses experimental autoimmune neuroinflammation by inducing antigen-specific Tregs

Lüth, S., Huber, S., Schramm, C., Buch, T., Zander, S., Stadelmann, C., Brück, W., Wraith, D. C., Herkel, J., & Lohse, A. W. (2008) · Journal of Clinical Investigation · doi:10.1172/jci32132 · Cited by 159 · see citing works

Novel Integrase-Defective Lentiviral Episomal Vectors for Gene Transfer

Vargas, J. J., Gusella, G., Najfeld, V., Klotman, M., & Cara, A. (2004) · Human Gene Therapy · doi:10.1089/104303404322959515 · Cited by 143 · see citing works

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

Annoni, A., Brown, B. D., Cantore, A., Sergi, L. S., Naldini, L., & Roncarolo, M. (2009) · Blood · doi:10.1182/blood-2009-04-214569 · Cited by 135 · see citing works

Integrase-defective lentiviral vectors: Progress and applications

Banasik, M. B., & McCray, P. B. (2010) · Gene Therapy · doi:10.1038/gt.2009.135 · Cited by 129 · see citing works

Unintegrated Lentivirus DNA Persistence and Accessibility to Expression in Nondividing Cells: Analysis with Class I Integrase Mutants

Saenz, D. T., Loewen, N. A., Peretz, M., Whitwam, T., Barraza, R., Howell, K. G., Holmes, J. M., Good, M. S., & Poeschla, E. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.6.2906-2920.2004 · Cited by 119 · see citing works

Hepatic Gene Transfer as a Means of Tolerance Induction to Transgene Products

LoDuca, P. A., Hoffman, B. E., & Herzog, R. W. (2009) · Current Gene Therapy · doi:10.2174/156652309787909490 · Cited by 114 · see citing works

Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection

Kantor, B., Ma, H., Webster-Cyriaque, J. Y., Monahan, P. E., & Kafri, T. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0905859106 · Cited by 98 · see citing works

Liver gene therapy: advances and hurdles

Nguyen, T., & Ferry, N. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302373 · Cited by 87 · see citing works

Successful Immunization with a Single Injection of Non-integrating Lentiviral Vector

Negri, D. R., Michelini, Z., Baroncelli, S., Spada, M., Vendetti, S., Buffa, V., Bona, R., Leone, P., Klotman, M. E., & Cara, A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300241 · Cited by 84 · see citing works

Protective Antiviral Immunity Conferred by a Nonintegrative Lentiviral Vector-Based Vaccine

Coutant, F., Frenkiel, M.-P., Despres, P., & Charneau, P. (2008) · PLoS ONE · doi:10.1371/journal.pone.0003973 · Cited by 70 · see citing works

Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment

Pierce, G. F., Lillicrap, D., Pipe, S. W., & VandenDriessche, T. (2007) · Journal of Thrombosis and Haemostasis · doi:10.1111/j.1538-7836.2007.02410.x · Cited by 56 · see citing works

Inverse vaccination, the opposite of Jenner’s concept, for therapy of autoimmunity

Steinman, L. (2010) · Journal of Internal Medicine · doi:10.1111/j.1365-2796.2010.02224.x · Cited by 44 · see citing works

IFN-α gene therapy for woodchuck hepatitis with adeno-associated virus: differences in duration of gene expression and antiviral activity using intraportal or intramuscular routes

Berraondo, P., Ochoa‐Callejero, L., Crettaz, J. S., Rotellar, F., Vales, Á., Martínez‐Ansó, E., Zaratiegui, M., Ruiz, J., González‐Aseguinolaza, G., & Prìeto, J. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.02.017 · Cited by 34 · see citing works

Gene therapy approaches to induce tolerance in autoimmunity by reshaping the immune system.

Wasserfall, C. H., & Herzog, R. W. (2009) · PubMed · Cited by 10 · see citing works

Immune Responses to Viral Gene Therapy Vectors

Shirley, J. L., de Jong, Y. P., Terhorst, C., & Herzog, R. W. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.01.001 · Cited by 788 · see citing works

Targeted genome editing in human repopulating haematopoietic stem cells

Genovese, P., Schiroli, G., Escobar, G., Di Tomaso, T., Firrito, C., Calabria, A., Moi, D., Mazzieri, R., Bonini, C., Holmes, M. C., et al. (2014) · Nature · doi:10.1038/nature13420 · Cited by 555 · see citing works

An unbiased genome-wide analysis of zinc-finger nuclease specificity

Gabriel, R., Lombardo, A., Arens, A., Miller, J. C., Genovese, P., Kaeppel, C., Nowrouzi, A., Bartholomae, C. C., Wang, J., Friedman, G., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1948 · Cited by 526 · see citing works

Site-specific integration and tailoring of cassette design for sustainable gene transfer

Lombardo, A., Cesana, D., Genovese, P., Stefano, B. D., Provasi, E., Colombo, D., Neri, M., Magnani, Z. I., Cantore, A., Riso, P. L., et al. (2011) · Nature Methods · doi:10.1038/nmeth.1674 · Cited by 329 · see citing works

TALEN-based Gene Correction for Epidermolysis Bullosa

Osborn, M. J., Starker, C. G., McElroy, A. N., Webber, B. R., Riddle, M. J., Xia, L., DeFeo, A. P., Gabriel, R., Schmidt, M., Von Kalle, C., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2013.56 · Cited by 258 · see citing works

Dynamics and genomic landscape of CD8+ T cells undergoing hepatic priming

Bénéchet, A. P., De Simone, G., Di Lucia, P., Cilenti, F., Barbiera, G., Le Bert, N., Fumagalli, V., Lusito, E., Moalli, F., Bianchessi, V., et al. (2019) · Nature · doi:10.1038/s41586-019-1620-6 · Cited by 222 · see citing works

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Cantore, A., Ranzani, M., Bartholomae, C. C., Volpin, M., Valle, P. D., Sanvito, F., Sergi, L. S., Gallina, P., Benedicenti, F., Bellinger, D., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aaa1405 · Cited by 158 · 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

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

Berdien, B., Mock, U., Atanackovic, D., & Fehse, B. (2014) · Gene Therapy · doi:10.1038/gt.2014.26 · Cited by 148 · see citing works

Next-generation AAV vectors—Do not judge a virus (only) by its cover

Domenger, C., & Grimm, D. (2019) · Human Molecular Genetics · doi:10.1093/hmg/ddz148 · Cited by 147 · see citing works

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

Milani, M., Annoni, A., Moalli, F., Liu, T., Cesana, D., Calabria, A., Bartolaccini, S., Biffi, M., Russo, F., Visigalli, I., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aav7325 · Cited by 142 · see citing works

Adenoviral vector DNA for accurate genome editing with engineered nucleases

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

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

Ferrari, S., Jacob, A., Cesana, D., Laugel, M., Beretta, S., Varesi, A., Unali, G., Conti, A., Canarutto, D., Albano, L., et al. (2022) · Cell stem cell · doi:10.1016/j.stem.2022.09.001 · Cited by 133 · see citing works

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

Du, L. M., Nurden, P., Nurden, A. T., Nichols, T. C., Bellinger, D. A., Jensen, E. S., Haberichter, S. L., Merricks, E., Raymer, R. A., Fang, J., et al. (2013) · Nature Communications · doi:10.1038/ncomms3773 · Cited by 119 · see citing works

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

Markusic, D. M., Hoffman, B. E., Perrin, G. Q., Nayak, S., Wang, X., LoDuca, P. A., High, K. A., & Herzog, R. W. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201302859 · Cited by 116 · see citing works

Fanconi Anemia Gene Editing by the CRISPR/Cas9 System

Osborn, M. J., Gabriel, R., Webber, B. R., DeFeo, A. P., McElroy, A. N., Jarjour, J., Starker, C. G., Wagner, J. E., Joung, J. K., Voytas, D. F., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2014.111 · Cited by 115 · see citing works

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

Maffioletti, S. M., Gerli, M., Ragazzi, M., Dastidar, S., Benedetti, S., Loperfido, M., VandenDriessche, T., Chuah, M., & Tedesco, F. S. (2015) · Nature Protocols · doi:10.1038/nprot.2015.057 · Cited by 114 · see citing works

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia b

Mingozzi, F., Chen, Y., Murphy, S. L., Edmonson, S. C., Tai, A., Price, S. D., Metzger, M. E., Zhou, S., Wright, J. F., Donahue, R. E., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.84 · Cited by 112 · see citing works

Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

Petrillo, C., Thorne, L. G., Unali, G., Schiroli, G., Giordano, A., Piras, F., Cuccovillo, I., Petit, S. J., Ahsan, F., Noursadeghi, M., et al. (2018) · Cell Stem Cell · doi:10.1016/j.stem.2018.10.008 · Cited by 102 · see citing works

Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice

Cantore, A., Nair, N., Della Valle, P., Di Matteo, M., Màtrai, J., Sanvito, F., Brombin, C., Di Serio, C., D'Angelo, A., Chuah, M., et al. (2012) · Blood · doi:10.1182/blood-2012-05-432591 · Cited by 101 · see citing works

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Dastidar, S., Ardui, S., Singh, K., Majumdar, D., Nair, N., Fu, Y., Reyon, D., Samara-Kuko, E., Gerli, M., Klein, A. F., et al. (2018) · Nucleic Acids Research · doi:10.1093/nar/gky548 · Cited by 96 · see citing works

MicroRNA-regulated viral vectors for gene therapy

Geisler, A., & Fechner, H. (2016) · World Journal of Experimental Medicine · doi:10.5493/wjem.v6.i2.37 · Cited by 96 · see citing works

Modulation of immune responses in lentiviral vector-mediated gene transfer

Annoni, A., Gregori, S., Naldini, L., & Cantore, A. (2018) · Cellular Immunology · doi:10.1016/j.cellimm.2018.04.012 · Cited by 89 · see citing works

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

Chuah, M., Petrus, I., Bleser, P. D., Guiner, C. L., Gernoux, G., Adjali, O., Nair, N., Willems, J., Evens, H., Rincón, M. Y., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.114 · Cited by 79 · see citing works

Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy

Tipanee, J., Samara-Kuko, E., Gevaert, T., Chuah, M. K., & VandenDriessche, T. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.06.006 · Cited by 76 · see citing works

Targeted gene therapy and cell reprogramming in Fanconi anemia

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

Genome editing for scalable production of alloantigen‐free lentiviral vectors for in vivo gene therapy

Milani, M., Annoni, A., Bartolaccini, S., Biffi, M., Russo, F., Di Tomaso, T., Raimondi, A., Lengler, J., Holmes, M. C., Scheiflinger, F., et al. (2017) · EMBO Molecular Medicine · doi:10.15252/emmm.201708148 · Cited by 72 · see citing works

Long Non-coding RNA DANCR as an Emerging Therapeutic Target in Human Cancers

Jin, S.-J., Jin, M., Xia, B.-R., & Jin, W. (2019) · Frontiers in Oncology · doi:10.3389/fonc.2019.01225 · Cited by 72 · see citing works

Development of gene transfer for induction of antigen-specific tolerance

Sack, B. K., Herzog, R. W., Terhorst, C., & Markusic, D. M. (2014) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1038/mtm.2014.13 · 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

Animal Models of Hemophilia

Sabatino, D. E., Nichols, T. C., Merricks, E. P., Bellinger, D. A., Herzog, R. W., & Monahan, P. E. (2011) · Progress in molecular biology and translational science · doi:10.1016/b978-0-12-394596-9.00006-8 · Cited by 67 · see citing works

Insulin B chain 9–23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3 + T regs

Akbarpour, M., Goudy, K., Cantore, A., Russo, F., Sanvito, F., Naldini, L., Annoni, A., & Roncarolo, M. G. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aaa3032 · Cited by 66 · see citing works

Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc‐/‐ mice

Lévy, C., Fusil, F., Amirache, F., Costa, C., Girard-Gagnepain, A., Négre, D., Bernadin, O., Garaulet, G., Rodrı́guez, A., Nair, N., et al. (2016) · Journal of Thrombosis and Haemostasis · doi:10.1111/jth.13520 · Cited by 66 · see citing works

CCN1 expression in hepatocytes contributes to macrophage infiltration in nonalcoholic fatty liver disease in mice

Bian, Z., Peng, Y., You, Z., Wang, Q., Miao, Q., Liu, Y., Han, X., Qiu, D., Li, Z., & Ma, X. (2012) · Journal of Lipid Research · doi:10.1194/jlr.m026013 · Cited by 64 · see citing works

Ethical and regulatory aspects of genome editing

Kohn, D. B., Porteus, M. H., & Scharenberg, A. M. (2016) · Blood · doi:10.1182/blood-2016-01-678136 · Cited by 67 · see citing works

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

Singh, K., Evens, H., Nair, N., Rincón, M. Y., Sarcar, S., Samara-Kuko, E., Chuah, M., & VandenDriessche, T. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.02.023 · Cited by 62 · 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

A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice

Merlin, S., Cannizzo, E. S., Borroni, E., Bruscaggin, V., Schinco, P., Tulalamba, W., Chuah, M., Arruda, V. R., VandenDriessche, T., Prat, M., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.04.029 · Cited by 60 · see citing works

Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice

Annoni, A., Cantore, A., Valle, P. D., Goudy, K., Akbarpour, M., Russo, F., Bartolaccini, S., D’Angelo, A., Roncarolo, M. G., & Naldini, L. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201302857 · Cited by 59 · see citing works

The challenges of promoting osteogenesis in segmental bone defects and osteoporosis

Ball, A. N., Donahue, S. W., Wojda, S. J., McIlwraith, C. W., Kawcak, C. E., Ehrhart, N. P., & Goodrich, L. R. (2017) · Journal of Orthopaedic Research® · doi:10.1002/jor.23845 · Cited by 59 · see citing works

Histone Deacetylase Inhibition Activates Transgene Expression from Integration-Defective Lentiviral Vectors in Dividing and Non-Dividing Cells

Pelascini, L. P. L., Janssen, J. M., & Gonçalves, M. A. F. V. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.069 · Cited by 57 · see citing works

Gene therapy for hemophilia

Herzog, R. W. (2015) · Frontiers in bioscience · doi:10.2741/4324 · Cited by 56 · see citing works

Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

Escobar, G., Barbarossa, L., Barbiera, G., Norelli, M., Genua, M., Ranghetti, A., Plati, T., Camisa, B., Brombin, C., Cittaro, D., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05315-0 · Cited by 54 · see citing works

Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy

Matteo, M. D., Samara-Kuko, E., Ward, N. J., Waddington, S. N., McVey, J. H., Chuah, M., & VandenDriessche, T. (2014) · Molecular Therapy · doi:10.1038/mt.2014.131 · Cited by 52 · see citing works

A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon

Burnight, E. R., Staber, J. M. R., Korsakov, P., Li, X., Brett, B. T., Scheetz, T. E., Craig, N. L., & McCray, P. B. (2012) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.12 · Cited by 49 · see citing works

Gene therapy for hemophilia

Chuah, M., Evens, H., & VandenDriessche, T. (2013) · Journal of Thrombosis and Haemostasis · doi:10.1111/jth.12215 · Cited by 48 · see citing works

A TLR and Non-TLR Mediated Innate Response to Lentiviruses Restricts Hepatocyte Entry and Can be Ameliorated by Pharmacological Blockade

Agudo, J., Ruzo, A., Kitur, K., Sachidanandam, R., Blander, J. M., & Brown, B. D. (2012) · Molecular Therapy · doi:10.1038/mt.2012.150 · Cited by 48 · see citing works

Role of the Vector Genome and Underlying Factor IX Mutation in Immune Responses to AAV Gene Therapy for Hemophilia B

Rogers, G. L., Martino, A. T., Zolotukhin, I., Ertl, H. C., & Herzog, R. W. (2014) · Journal of Translational Medicine · doi:10.1186/1479-5876-12-25 · Cited by 45 · see citing works

Emerging and future therapies for hemophilia

Carr, M. E., & Tortella, B. J. (2015) · Journal of Blood Medicine · doi:10.2147/jbm.s42669 · Cited by 45 · see citing works

Hemophilia Gene Therapy: The End of the Beginning?

Wolf, D. D., Singh, K., Chuah, M., & VandenDriessche, T. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.112 · Cited by 45 · see citing works

Bioinformatic Clonality Analysis of Next-Generation Sequencing-Derived Viral Vector Integration Sites

Arens, A., Appelt, J.-U., Bartholomae, C. C., Gabriel, R., Paruzynski, A., Gustafson, D., Cartier, N., Aubourg, P. R., Deichmann, A., Glimm, H., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2011.219 · Cited by 45 · see citing works

Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy

Rincón, M. Y., Sarcar, S., Danso‐Abeam, D., Keyaerts, M., Mátrai, J., Samara-Kuko, E., Acosta‐Sanchez, A., Athanasopoulos, T., Dickson, G., Lahoutte, T., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.178 · Cited by 45 · see citing works

Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice

Suwanmanee, T., Hu, G., Gui, T., Bartholomae, C. C., Kutschera, I., Kalle, C. V., Schmidt, M., Monahan, P. E., & Kafri, T. (2013) · Molecular Therapy · doi:10.1038/mt.2013.188 · Cited by 44 · see citing works

Dynamics of antigen presentation to transgene product-specific CD4+ T cells and of Treg induction upon hepatic AAV gene transfer

Perrin, G. Q., Zolotukhin, I., Sherman, A., Biswas, M., de Jong, Y. P., Terhorst, C., Davidoff, A. M., & Herzog, R. W. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.83 · Cited by 40 · see citing works

Long-Term Episomal Transgene Expression from Mitotically Stable Integration-Deficient Lentiviral Vectors

Kymäläinen, H., Appelt, J. U., Giordano, F. A., Davies, A. F., Ogilvie, C. M., Ahmed, S., Laufs, S., Schmidt, M., Bode, J., Yáñez‐Muñoz, R. J., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.172 · Cited by 38 · see citing works

Prolonged Prophylactic Protection from Botulism with a Single Adenovirus Treatment Promoting Serum Expression of a VHH-Based Antitoxin Protein

Mukherjee, J., Dmitriev, I., Debatis, M., Tremblay, J. M., Beamer, G., Kashentseva, E. A., Curiel, D. T., & Shoemaker, C. B. (2014) · PLoS ONE · doi:10.1371/journal.pone.0106422 · Cited by 37 · see citing works

Exploiting microRNA regulation for genetic engineering

Gentner, B., & Naldini, L. (2012) · Tissue Antigens · doi:10.1111/tan.12002 · Cited by 35 · see citing works

Liver‐targeted gene therapy: Approaches and challenges

Aravalli, R. N., Belcher, J. D., & Steer, C. J. (2015) · Liver Transplantation · doi:10.1002/lt.24122 · Cited by 35 · see citing works

Integrase-Deficient Lentiviral Vectors Mediate Efficient Gene Transfer to Human Vascular Smooth Muscle Cells with Minimal Genotoxic Risk

Chick, H., Nowrouzi, A., Fronza, R., Mcdonald, R., Kane, N., Alba, R., Delles, C., Sessa, W., Schmidt, M., Thrasher, A., et al. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.042 · Cited by 19 · see citing works

Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A

Sherman, A., Biswas, M., & Herzog, R. W. (2017) · Frontiers in Immunology · doi:10.3389/fimmu.2017.01604 · Cited by 33 · see citing works

New approaches to gene and cell therapy for hemophilia

Ohmori, T., Mizukami, H., OZAWA, K., Sakata, Y., & Nishimura, S. (2015) · Journal of Thrombosis and Haemostasis · doi:10.1111/jth.12926 · Cited by 32 · see citing works

A Nonintegrative Lentiviral Vector-Based Vaccine Provides Long-Term Sterile Protection against Malaria

Coutant, F., Sanchez David, R. Y., Félix, T., Boulay, A., Caleechurn, L., Souque, P., Thouvenot, C., Bourgouin, C., Beignon, A.-S., & Charneau, P. (2012) · PLoS ONE · doi:10.1371/journal.pone.0048644 · Cited by 32 · see citing works

New Frontier in Regenerative Medicine: Site-Specific Gene Correction in Patient-Specific Induced Pluripotent Stem Cells

Garate, Z., Davis, B. R., Bustamante, O. Q., & Segovia, J. C. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.251 · Cited by 31 · see citing works

Cell-based therapies for Huntington's disease

Chen, Y., Carter, R. L., Cho, I. K., & Chan, A. W. S. (2014) · Drug Discovery Today · doi:10.1016/j.drudis.2014.02.012 · Cited by 31 · see citing works

Hemophilia Gene Therapy: Ready for Prime Time?

VandenDriessche, T., & Chuah, M. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.116 · Cited by 31 · see citing works

Recent Progress in Gene Therapy for Hemophilia

Chuah, M., Nair, N., & VandenDriessche, T. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.088 · Cited by 29 · see citing works

Cellular Immune Response Against Firefly Luciferase After Sleeping Beauty –Mediated Gene Transfer In Vivo

Podetz-Pedersen, K. M., Vezys, V., Somia, N. V., Russell, S. J., & McIvor, R. S. (2014) · Human Gene Therapy · doi:10.1089/hum.2014.048 · Cited by 29 · see citing works

Integration of retroviral vectors

Gabriel, R., Schmidt, M., & Kalle, C. V. (2012) · Current Opinion in Immunology · doi:10.1016/j.coi.2012.08.006 · Cited by 29 · see citing works

Immune responses in liver-directed lentiviral gene therapy

Annoni, A., Goudy, K., Akbarpour, M., Naldini, L., & Roncarolo, M. G. (2013) · Translational research · doi:10.1016/j.trsl.2012.12.018 · Cited by 30 · see citing works

Integration-deficient lentivectors: an effective strategy to purify and differentiate human embryonic stem cell-derived hepatic progenitors

Yang, G., Si-Tayeb, K., Corbineau, S., Vernet, R., Gayon, R., Dianat, N., Martinet, C., Clay, D., Goulinet-Mainot, S., Tachdjian, G., et al. (2013) · BMC Biology · doi:10.1186/1741-7007-11-86 · Cited by 26 · see citing works

Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents

Rittelmeyer, I., Rothe, M., Brugman, M. H., Iken, M., Schambach, A., Manns, M. P., Baum, C., Modlich, U., & Ott, M. (2012) · Hepatology · doi:10.1002/hep.26204 · Cited by 26 · see citing works

New Methods for Disease Modeling Using Lentiviral Vectors

Alfranca, A., Campanero, M. R., & Redondo, J. M. (2018) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2018.08.001 · Cited by 26 · see citing works

Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors

Merlin, S., & Follenzi, A. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.12.013 · Cited by 25 · see citing works

Haemophilia gene therapy: From trailblazer to gamechanger

Evens, H., Chuah, M., & VandenDriessche, T. (2018) · Haemophilia · doi:10.1111/hae.13494 · Cited by 23 · see citing works

Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives

Zhou, W., Yang, J., Zhang, Y., Hu, X., & Wang, W. (2022) · MedComm · doi:10.1002/mco2.155 · Cited by 22 · see citing works

The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis

Nowrouzi, A., Cheung, W. T., Li, T., Zhang, X., Arens, A., Paruzynski, A., Waddington, S. N., Osejindu, E., Reja, S., Kalle, C. V., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2012.224 · Cited by 22 · see citing works

Gene editing technology as an approach to the treatment of liver diseases

Aravalli, R. N., & Steer, C. J. (2016) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2016.1158808 · Cited by 22 · see citing works

Transgenic Expression of Human Glial Cell Line-Derived Neurotrophic Factor from Integration-Deficient Lentiviral Vectors is Neuroprotective in a Rodent Model of Parkinson's Disease

Lu-Nguyen, N. B., Broadstock, M., Schliesser, M. G., Bartholomae, C., von Kalle, C., Schmidt, M., & Yáñez-Muñoz, R. (2014) · Human Gene Therapy · doi:10.1089/hum.2014.003 · Cited by 22 · see citing works

Simian immunodeficiency virus-Vpx for improving integrase defective lentiviral vector-based vaccines

Negri, D. R. M., Rossi, A., Blasi, M., Michelini, Z., Leone, P., Chiantore, M. V., Baroncelli, S., Perretta, G., Cimarelli, A., Klotman, M. E., et al. (2012) · Retrovirology · doi:10.1186/1742-4690-9-69 · Cited by 22 · see citing works

In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking

Milani, M., Fabiano, A., Perez-Rodriguez, M., Hernández, R. J., Zecchillo, A., Zonari, E., Ottonello, S., Ricci, L. B., Canepari, C., Volpin, M., et al. (2025) · Nature · doi:10.1038/s41586-025-09070-3 · Cited by 20 · see citing works

Integrase-defective lentiviral vectors encoding cytokines induce differentiation of human dendritic cells and stimulate multivalent immune responses in vitro and in vivo

Daenthanasanmak, A., Salguero, G., Borchers, S., Figueiredo, C., Jacobs, R., Sundarasetty, B. S., Schneider, A., Schambach, A., Eiz‐Vesper, B., Blasczyk, R., et al. (2012) · Vaccine · doi:10.1016/j.vaccine.2012.05.063 · Cited by 20 · see citing works

TraFo-CRISPR: Enhanced Genome Engineering by Transient Foamy Virus Vector-Mediated Delivery of CRISPR/Cas9 Components

Lindel, F., Dodt, C., Weidner, N., Noll, M., Bergemann, F., Behrendt, R., Fischer, S., Dietrich, J., Cartellieri, M., Hamann, M. V., et al. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.10.006 · Cited by 20 · see citing works

Perinatal Gene Transfer to the Liver

McKay, T. R., Rahim, A. A., Buckley, S. M. K., Ward, N. J., Chan, J. K. Y., Howe, S. J., & Waddington, S. N. (2011) · Current Pharmaceutical Design · doi:10.2174/138161211797247541 · Cited by 20 · see citing works

GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice

Milani, M., Canepari, C., Assanelli, S., Merlin, S., Borroni, E., Starinieri, F., Biffi, M., Russo, F., Fabiano, A., Zambroni, D., et al. (2024) · EMBO Molecular Medicine · doi:10.1038/s44321-024-00072-8 · Cited by 18 · see citing works

CRISPR/Cas9 therapeutics for liver diseases

Aravalli, R. N., & Steer, C. J. (2017) · Journal of Cellular Biochemistry · doi:10.1002/jcb.26627 · Cited by 18 · see citing works

Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors

Singh, K., Fronza, R., Evens, H., Chuah, M., & VandenDriessche, T. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101365 · Cited by 19 · see citing works

Generation of lentivirus-induced dendritic cells under GMP-compliant conditions for adaptive immune reconstitution against cytomegalovirus after stem cell transplantation

Sundarasetty, B. S., Kloeß, S., Oberschmidt, O., Naundorf, S., Kuehlcke, K., Daenthanasanmak, A., Gerasch, L., Figueiredo, C., Blasczyk, R., Ruggiero, E., et al. (2015) · Journal of Translational Medicine · doi:10.1186/s12967-015-0599-5 · Cited by 17 · see citing works

Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy

Goudy, K., Annoni, A., Naldini, L., & Roncarolo, M. (2011) · Frontiers in Microbiology · doi:10.3389/fmicb.2011.00221 · Cited by 18 · see citing works

Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes

Canepari, C., Milani, M., Simoni, C., Starinieri, F., Volpin, M., Fabiano, A., Biffi, M., Russo, F., Norata, R., Rocchi, M., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-60073-0 · Cited by 16 · see citing works

Comparison Between Several Integrase-defective Lentiviral Vectors Reveals Increased Integration of an HIV Vector Bearing a D167H Mutant

Saeed, M. Q., Dufour, N., Bartholmae, C., Sieranska, U., Knopf, M., Thierry, E., Thierry, S., Delelis, O., Grandchamp, N., Pilet, H., et al. (2014) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2014.65 · Cited by 14 · see citing works

Protection is not always a good thing: The immune system’s impact on gene therapy

de Freitas, M. V., Frâncio, L., Haleva, L., & Matte, Ú. D. S. (2022) · Genetics and Molecular Biology · doi:10.1590/1678-4685-gmb-2022-0046 · Cited by 16 · see citing works

Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors

Hu, P., Li, Y., Sands, M. S., McCown, T. J., & Kafri, T. (2015) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.25 · Cited by 15 · see citing works

Targeting endothelial cells by gene therapy

VandenDriessche, T., & Chuah, M. (2013) · Blood · doi:10.1182/blood-2013-08-518266 · Cited by 14 · see citing works

Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX

Shi, Y., Falahati, R., Zhang, J., Flebbe-Rehwaldt, L., & Gaensler, K. M. L. (2013) · Gene Therapy · doi:10.1038/gt.2013.22 · Cited by 13 · see citing works

Linear Amplification Mediated PCR – Localization of Genetic Elements and Characterization of Unknown Flanking DNA

Gabriel, R., Kutschera, I., Bartholomae, C. C., Kalle, C. V., & Schmidt, M. (2014) · Journal of Visualized Experiments · doi:10.3791/51543 · Cited by 14 · see citing works

Intrathymic injection of lentiviral vector curtails the immune response in the periphery of normal mice

Gottrand, G., Taleb, K., Ragon, I., Bergot, A., Goldstein, J. D., & Marodon, G. (2012) · The Journal of Gene Medicine · doi:10.1002/jgm.1650 · Cited by 13 · see citing works

The Evolution of Gene Therapy in the Treatment of Metabolic Liver Diseases

Moscoso, C. G., & Steer, C. J. (2020) · Genes · doi:10.3390/genes11080915 · Cited by 12 · see citing works

Progress and Problems with Viral Vectors for Delivery of Talens

Bergmann, T. (2014) · Journal of Molecular and Genetic Medicine · doi:10.4172/1747-0862.1000096 · Cited by 11 · see citing works

InsB9-23 Gene Transfer to Hepatocyte-Based Combined Therapy Abrogates Recurrence of Type 1 Diabetes After Islet Transplantation

Russo, F., Citro, A., Squeri, G., Sanvito, F., Monti, P., Gregori, S., Roncarolo, M. G., & Annoni, A. (2020) · Diabetes · doi:10.2337/db19-1249 · Cited by 11 · see citing works

In Vivo Gene Delivery to Lymph Node Stromal Cells Leads to Transgene-specific CD8+ T Cell Anergy in Mice

Ciré, S., Rocha, S. D., Ferrand, M., Collins, M. K., & Galy, A. H. M. (2016) · Molecular Therapy · doi:10.1038/mt.2016.168 · Cited by 10 · see citing works

Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance

Mátrai, J., Cantore, A., Bartholomae, C. C., Annoni, A., Wang, W., Acosta‐Sanchez, A., Samara-kuko, E., Waele, L. D., Ma, L., Genovese, P., et al. (2013)

377. Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Regulatory T Cells and Immune Tolerance to Foreign Antigens

(2011) · Molecular Therapy · doi:10.1016/s1525-0016(16)36949-0

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

Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

Follenzi, A., Battaglia, M., Lombardo, A., Annoni, A., Roncarolo, M. G., & Naldini, L. (2004) · Blood · doi:10.1182/blood-2003-09-3217 · Cited by 212 · see citing works

Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression <i>in vitro</i> and <i>in vivo</i>

Philippe, S., Sarkis, C., Barkats, M., Mammeri, H., Ladroue, C., Petit, C., Mallet, J., & Serguera, C. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0606197103 · Cited by 249 · see citing works