The DOI matches its Crossref record; 45 of the 45 references it cites were found in a registry or an index.
Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy
Abstract
Gene therapy is a promising therapeutic approach to treat primary immunodeficiencies. Indeed, the clinical trial for the Wiskott-Aldrich Syndrome (WAS) that is currently ongoing at the Hannover Medical School (Germany) has recently reported the correction of all affected cell lineages of the hematopoietic system in the first treated patients. However, an extensive study of the clonal inventory of those patients reveals that LMO2, CCND2 and MDS1/EVI1 were preferentially prevalent. Moreover, a first leukemia case was observed in this study, thus reinforcing the need of developing safer vectors for gene transfer into HSC in general. Here we present a novel self-inactivating (SIN) vector for the gene therapy of WAS that combines improved safety features. We used the elongation factor 1 alpha (EFS) promoter, which has been extensively evaluated in terms of safety profile, to drive a codon-optimized human WASP cDNA. To test vector performance in a more clinically relevant setting, we transduced murine HSPC as well as human CD34+ cells and also analyzed vector efficacy in their differentiated myeloid progeny. Our results show that our novel vector generates comparable WAS protein levels and is as effective as the clinically used LTR-driven vector. Therefore, the described SIN vectors appear to be good candidates for potential use in a safer new gene therapy protocol for WAS, with decreased risk of insertional mutagenesis.
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“There have been concerns about mutagenesis related to viral integration after the addition of a gene to hematopoietic stem cells ever since leukemia and the myelodysplastic syndrome developed in patients who were treated with gamma-retro-viral vector gene therapy for several immunodeficiency diseases.25–28”
From Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy (2017) · cites this paper as [28]
Citations
Cites
A new mathematical model for relative quantification in real-time RT-PCR
Pfaffl, M. W. (2001) · Nucleic Acids Research · doi:10.1093/nar/29.9.e45 · Cited by 34,610
A Third-Generation Lentivirus Vector with a Conditional Packaging System
Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341
Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
Cavazzana-Calvo, M., Hacein-Bey, S., Basile, G. D. S., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.-L., et al. (2000) · Science · doi:10.1126/science.288.5466.669 · Cited by 2,167
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
HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots
Schröder, A. R. W., Shinn, P., Chen, H., Berry, C., Ecker, J. R., & Bushman, F. (2002) · Cell · doi:10.1016/s0092-8674(02)00864-4 · Cited by 1,832
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
Ott, M. G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kühlcke, K., Schilz, A., Kunkel, H., et al. (2006) · Nature Medicine · doi:10.1038/nm1393 · Cited by 1,051
Isolation of a novel gene mutated in Wiskott-Aldrich syndrome
Derry, J. M. J., Ochs, H. D., & Francke, U. (1994) · Cell · doi:10.1016/0092-8674(94)90528-2 · Cited by 1,043
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) · Science · doi:10.1126/science.1070104 · Cited by 975
Stein, S., Ott, M. G., Schultze-Strasser, S., Jauch, A., Burwinkel, B., Kinner, A., Schmidt, M., Krämer, A., Schwäble, J., Glimm, H., et al. (2010) · Nature Medicine · doi:10.1038/nm.2088 · Cited by 716
A multiinstitutional survey of the Wiskott-Aldrich syndrome
Sullivan, K. E., Mullen, C. A., Blaese, R. M., & Winkelstein, J. A. (1994) · The Journal of Pediatrics · doi:10.1016/s0022-3476(05)82002-5 · Cited by 636
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
Prospects for Human Gene Therapy
Anderson, W. F. (1984) · Science · doi:10.1126/science.6093246 · Cited by 533
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
Self-citationOchs, H. D., & Thrasher, A. J. (2006) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2006.02.005 · Cited by 437
Wiskott-Aldrich syndrome protein regulates podosomes in primary human macrophages
Linder, S., Nelson, D., Weiss, M., & Aepfelbacher, M. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.17.9648 · Cited by 430
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
Self-citationReproducible establishment of hemopoietic supportive stromal cell lines from murine bone marrow
伊藤, 克. (1990) · Medical Entomology and Zoology · Cited by 309
Cell culture assays reveal the importance of retroviral vector design for insertional genotoxicity
Modlich, U., Bohne, J., Schmidt, M., von Kalle, C., Knöss, S., Schambach, A., & Baum, C. (2006) · Blood · doi:10.1182/blood-2005-08-024976 · Cited by 278
Self-citationPhysiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors
Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., & Baum, C. (2008) · Molecular Therapy · doi:10.1038/mt.2008.5 · Cited by 271
Self-citationHematti, P., Hong, B.-K., Ferguson, C., Adler, R. L., Hanawa, H., Sellers, S. E., Holt, I. E., Eckfeldt, C. E., Sharma, Y., Schmidt, M., et al. (2004) · PLoS Biology · doi:10.1371/journal.pbio.0020423 · Cited by 269
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 195
Self-citationThornhill, S. I., Schambach, A., Howe, S. J., Ulaganathan, M., Grassman, E., Williams, D. A., Schiedlmeier, B., Sebire, N. J., Gaspar, H. B., Kinnon, C., et al. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300393 · Cited by 166
Self-citationSchambach, 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 148
Self-citationSchambach, 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 147
Self-citationMechanisms of WASp-mediated hematologic and immunologic disease
Burns, S. O., Cory, G. O. C., Vainchenker, W., et al. (2004) · Blood · doi:10.1182/blood-2004-04-1678 · Cited by 146
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
Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center
Ozsahin, H., Deist, F. L., Benkerrou, M., Cavazzana, M., Gomez, L., Griscelli, C., Blanche, S., & Fischer, A. (1996) · The Journal of Pediatrics · doi:10.1016/s0022-3476(96)70248-2 · Cited by 97
Charrier, S., Dupré, L., Scaramuzza, S., Jeanson-Leh, L., Blundell, M. P., Danos, O., Cattaneo, F., Aiuti, A., Eckenberg, R., Thrasher, A. J., et al. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302863 · Cited by 91
Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., Panaroni, C., Ponzoni, M., Sanvito, F., Doglioni, C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.31 · Cited by 85
Maruggi, G., Porcellini, S., Facchini, G., Perna, S. K., Cattoglio, C., Sartori, D., Ambrosi, A., Schambach, A., Baum, C., Bonini, C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.51 · Cited by 81
Self-citationMoreno-Carranza, B., Gentsch, M., Stein, S., Schambach, A., Santilli, G., Rudolf, E., Ryser, M., Haria, S., Thrasher, A. J., Baum, C., et al. (2008) · Gene Therapy · doi:10.1038/gt.2008.143 · Cited by 61
Self-citationPreventing and exploiting the oncogenic potential of integrating gene vectors
Modlich, U., & Baum, C. (2009) · Journal of Clinical Investigation · doi:10.1172/jci38831 · Cited by 61
Self-citationDevelopment of lentiviral gene therapy for Wiskott Aldrich syndrome
Galy, A. H. M., Roncarolo, M., & Thrasher, A. J. (2008) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.8.2.181 · Cited by 59
Polyclonal fluctuation of lentiviral vector–transduced and expanded murine hematopoietic stem cells
Maetzig, T., Brugman, M. H., Bartels, S., Heinz, N., Kustikova, O. S., Modlich, U., Li, Z., Galla, M., Schiedlmeier, B., Schambach, A., et al. (2011) · Blood · doi:10.1182/blood-2010-08-303222 · Cited by 56
Self-citationA Partial Down-regulation of WASP Is Sufficient to Inhibit Podosome Formation in Dendritic Cells
Olivier, A., Jeanson, L., Bouma, G., Compagno, D., Blondeau, J., Seye, K., Charrier, S., Burns, S. O., Thrasher, A. J., Danos, O., et al. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.003 · Cited by 55
Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Boztuǧ, K., Dewey, R. A., & Klein, C. (2006) · PubMed · Cited by 47
Self-citationNg, Y. Y., Baert, M. R. M., Pike‐Overzet, K., Rodijk, M., Brugman, M. H., Schambach, A., Baum, C., Hendriks, R. W., van Dongen, J. J. M., & Staal, F. J. T. (2010) · Leukemia · doi:10.1038/leu.2010.140 · Cited by 43
Self-citationHuston, M. W., van Til, N. P., Visser, T. P., Arshad, S., Brugman, M. H., Cattoglio, C., Nowrouzi, A., Li, Y., Schambach, A., Schmidt, M., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.127 · Cited by 41
Self-citationImprovement of Migratory Defects in a Murine Model of Wiskott–Aldrich Syndrome Gene Therapy
Blundell, M. P., Bouma, G., Calle, Y., Jones, G. E., Kinnon, C., & Thrasher, A. J. (2008) · Molecular Therapy · doi:10.1038/mt.2008.43 · Cited by 39
Design and Production of Retro- and Lentiviral Vectors for Gene Expression in Hematopoietic Cells
Schambach, A., Swaney, W. P., & van der Loo, J. C. M. (2009) · Methods in molecular biology · doi:10.1007/978-1-59745-409-4_14 · Cited by 38
Self-citationDewey, R. A., Díez, I. A., Ballmaier, M., Filipovich, A. H., Greil, J., Güngör, T., Happel, C. M., Maschan, A., Noyan, F., Pannicke, U., et al. (2006) · Experimental Hematology · doi:10.1016/j.exphem.2006.04.021 · Cited by 37
Self-citationSolving the Problem of γ-Retroviral Vectors Containing Long Terminal Repeats
Persons, D. A., & Baum, C. (2011) · Molecular Therapy · doi:10.1038/mt.2010.305 · Cited by 34
Self-citationA Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice
Metcalf, J. A., Ma, X., Linders, B., Wu, S. J., Schambach, A., Ohlemiller, K. K., Kovács, A., Bigg, M., He, L., Tollefsen, D. M., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.236 · Cited by 29
Self-citation
Research works printed but not linked: 4
- Boztug K., 2006, Curr. Opin. Mol. Ther., 8, 390
- Itoh K., 1989, Exp. Hematol., 17, 145
- IDW Press Release:Effective gene therapy for children with Wiskott-Aldrich-syndrome. Nov 11, 2010.
- Modlich U., 2009, Mol. Ther., 17, 1919, 10.1038/mt.2009.179 · DOI · Paper page
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Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1700554 · Cited by 604
Negre, O., Bartholomae, C., Beuzard, Y., Cavazzana, M., Christiansen, L., Courne, C., Deichmann, A., Denaro, M., Dreuzy, E., Finer, M., et al. (2014) · Current Gene Therapy · doi:10.2174/1566523214666141127095336 · Cited by 126
Retroviral Integrations in Gene Therapy Trials
Biasco, L., Baricordi, C., & Aiuti, A. (2012) · Molecular Therapy · doi:10.1038/mt.2011.289 · Cited by 106
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
Cavazzana, M., Six, E., Lagresle-Peyrou, C., André-Schmutz, I., & Hacein-Bey-Abina, S. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.137 · Cited by 106
Williams, D. A., & Thrasher, A. J. (2014) · Stem Cells Translational Medicine · doi:10.5966/sctm.2013-0206 · Cited by 56
Metadata
- Authors
- Avedillo Díez, I., Zychlinski, D., Coci, E. G., Galla, M., Modlich, U., Dewey, R. A., Schwarzer, A., Maetzig, T., Mpofu, N., Jaeckel, E., Boztug, K., Baum, C., Klein, C., & Schambach, A.
- Affiliations
- Department of Pediatric Hematology/Oncology, ∥Institute of Experimental Hematology, ‡Excellence Cluster REBIRTH, ⊥Integrated Research and Treatment Center Transplantation, and #Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Germany
- Venue
- Molecular Pharmaceutics
- Publisher
- American Chemical Society (ACS)
- Published
- 2011-08-31
- Volume
- 8
- Issue
- 5
- Pages
- 1525-1537
- ISSN
- 1543-8384
- 1543-8392
- Cited by
- 59
- References
- 45
- Language
- en
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