Development of Gene Therapy for Thalassemia

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

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<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

Position-independent, high-level expression of the human β-globin gene in transgenic mice

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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

HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots

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Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery

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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

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

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

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

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

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

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

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) · Science · doi:10.1126/science.1070104 · Cited by 1,137 · 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

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

Aiuti, A., Cattaneo, F., Galimberti, S., Benninghoff, U., Cassani, B., Callegaro, L., Scaramuzza, S., Andolfi, G., Mirolo, M., Brigida, I., et al. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa0805817 · Cited by 1,003 · see citing works

Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

Follenzi, A., Ailles, L., Bakovic, S., Geuna, M., & Naldini, L. (2000) · Nature Genetics · doi:10.1038/76095 · Cited by 926 · see citing works

HIV-1 genome nuclear import is mediated by a central DNA flap.

Zennou, V., Petit, C., Guétard, D., Nerhbass, U., Montagnier, L., & Charneau, P. (2000) · Cell · doi:10.1016/s0092-8674(00)80828-4 · Cited by 893 · see citing works

A 5′ element of the chicken β-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila

Chung, J. H., Whiteley, M., & Felsenfeld, G. (1993) · Cell · doi:10.1016/0092-8674(93)80052-g · Cited by 854 · see citing works

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

Stein, S., Ott, M., Schultze-Strasser, S., Jauch, A., Burwinkel, B., Kinner, A., Schmidt, M., Krämer, A., Schwable, J., Glimm, H., et al. (2010) · Nature Medicine · doi:10.1038/nm.2088 · Cited by 783 · see citing works

Preferential associations between co-regulated genes reveal a transcriptional interactome in erythroid cells

Schoenfelder, S., Sexton, T., Chakalova, L., Cope, N. F., Horton, A., Andrews, S., Kurukuti, S., Mitchell, J. A., Umlauf, D., Dimitrova, D. S., et al. (2009) · Nature Genetics · doi:10.1038/ng.496 · Cited by 710 · 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

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein‐Bey‐Abina, S., Hauer, J., Lim, A., Pïcard, C., Wang, G. P., Berry, C. C., Martinache, C., Rieux‐Laucat, F., Latour, S., Belohradsky, B. H., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000164 · Cited by 637 · see citing works

The "beta-like-globin" gene domain in human erythroid cells.

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

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

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

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

Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells.

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Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

Transgenic Knockout Mice with Exclusively Human Sickle Hemoglobin and Sickle Cell Disease

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Evidence for a locus activation region: the formation of developmentally stable hypersensitive sites in globin-expressing hybrids

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Loss of transcriptional activity of a transgene is accompanied by DNA methylation and histone deacetylation and is prevented by insulatorsPreprint

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Characterization of the chicken β-globin insulator

Chung, J. H., Bell, A. C., & Felsenfeld, G. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.2.575 · Cited by 393 · 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

Stable transduction of quiescent CD34 + CD38 − human hematopoietic cells by HIV-1-based lentiviral vectors

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Position-effect protection and enhancer blocking by the chicken β-globin insulator are separable activities

Targa, F. R., Pikaart, M. J., Burgess-Beusse, B. L., Bell, A. C., Litt, M. D., West, A. G., Gaszner, M., & Felsenfeld, G. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.102179399 · Cited by 381 · see citing works

Synthesis of rabbit β-globin in cultured monkey kidney cells following infection with a SV40 β-globin recombinant genome

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Lineage-specific expression of a human β-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells

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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

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

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

Papapetrou, E. P., Lee, G., Malani, N., Setty, M. N., Rivière, I., Tirunagari, L. M., Kadota, K., Roth, S. L., Giardina, P. J., Viale, A. J., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1717 · Cited by 300 · see citing works

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

Gaspar, H., Parsley, K., Howe, S., King, D., Gilmour, K. C., Sinclair, J., Brouns, G., Schmidt, M., von Kalle, C., Barington, T., et al. (2004) · The Lancet · doi:10.1016/s0140-6736(04)17590-9 · Cited by 300 · see citing works

HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G 0 /G 1 human hematopoietic stem cells

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Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

Gaspar, H. B., Cooray, S., Gilmour, K. C., Parsley, K. L., Zhang, F., Adams, S. P., Bjorkegren, E., Xu‐Bayford, J., Brown, L., Davies, E. G., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3002716 · Cited by 284 · see citing works

A mouse model for beta 0-thalassemia.

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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

Recruitment of Histone Modifications by USF Proteins at a Vertebrate Barrier Element

West, A. G., Huang, S., Gaszner, M., Litt, M. D., & Felsenfeld, G. (2004) · Molecular Cell · doi:10.1016/j.molcel.2004.10.005 · Cited by 258 · see citing works

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

Deichmann, A., Hacein‐Bey‐Abina, S., Schmidt, M., Garrigue, A., Brugman, M. H., Hu, J., Glimm, H., Gyapay, G., Prum, B., Fraser, C. C., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci31659 · Cited by 257 · see citing works

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

Aiuti, A., Cassani, B., Andolfi, G., Mirolo, M., Biasco, L., Recchia, A., Urbinati, F., Valacca, C., Scaramuzza, S., Aker, M., et al. (2007) · Journal of Clinical Investigation · doi:10.1172/jci31666 · Cited by 252 · 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

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

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The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells.

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Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency

Gaspar, H. B., Cooray, S., Gilmour, K. C., Parsley, K. L., Adams, S. P., Howe, S. J., Ghonaium, A. A., Xu‐Bayford, J., Brown, L., Davies, E. G., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3002715 · Cited by 231 · see citing works

The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells

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A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer.

Rivella, S., May, C., Chadburn, A., Rivière, I., & Sadelain, M. (2003) · Blood · doi:10.1182/blood-2002-10-3305 · Cited by 220 · see citing works

Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities

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

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Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells.

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Nicotinamide, a SIRT1 inhibitor, inhibits differentiation and facilitates expansion of hematopoietic progenitor cells with enhanced bone marrow homing and engraftment

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Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene.

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Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

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Transduction of Human Progenitor Hematopoietic Stem Cells by Human Immunodeficiency Virus Type 1-Based Vectors Is Cell Cycle Dependent

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Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells

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Successful correction of the human β-thalassemia major phenotype using a lentiviral vector

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Enhancers and silencers: an integrated and simple model for their function

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A Mouse Model for b-Thalassemia

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Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency

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Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene

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Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

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Mutagenesis of retroviral vectors transducing human beta‐globin gene and beta‐globin locus control region derivatives results in stable transmission of an active transcriptional structure.

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The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number

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Critical Factors Influencing Stable Transduction of Human CD34+ Cells with HIV-1-Derived Lentiviral Vectors

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Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector.

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Chapter 4 β‐Globin Regulation and Long‐Range Interactions

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

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Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

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Transcription factories: genetic programming in three dimensions

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Failure of SCID-X1 gene therapy in older patients

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High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells.

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Retroviral Integrations in Gene Therapy Trials

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