Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.
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Christine Kinnon | Christof von Kalle | Michael Hubank | Susannah I Thornhill | Stuart Adams | Dick de Ridder | Manfred Schmidt | Karin Pike-Overzet | Michelle Quaye | Kerstin Schwarzwaelder | Frank J T Staal | Philip Ancliff | Kathryn L. Parsley | C. von Kalle | F. Staal | M. Hubank | D. de Ridder | D. Linch | A. Thrasher | Manfred Schmidt | K. Pike-Overzet | C. Bartholomae | S. Howe | C. Kinnon | H. Kempski | K. Gilmour | H. Gaspar | M. Brugman | M. Mansour | Adrian J Thrasher | P. Ancliff | R. Gale | S. Thornhill | Cynthia Bartholomae | H Bobby Gaspar | Helena Kempski | Marc R Mansour | Martijn H Brugman | Rosemary E Gale | David C Linch | Steven J Howe | Kathryn L Parsley | Kimberly C Gilmour | Jinhua Bayford | Stephen J Chatters | Lucie Brown | David K Webb | S. Adams | K. Parsley | S. Chatters | Kerstin Schwarzwaelder | D. K. Webb | J. Bayford | L. Brown | M. Quaye | D. Webb | M. Schmidt
[1] Andrew P. Weng,et al. Activating Mutations of NOTCH1 in Human T Cell Acute Lymphoblastic Leukemia , 2004, Science.
[2] L. Notarangelo,et al. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement , 2002, Nature Medicine.
[3] W. Leonard,et al. Interleukin-2 receptor γ chain mutation results in X-linked severe combined immunodeficiency in humans , 1993, Cell.
[4] N. Copeland,et al. Gene Therapy Insertional Mutagenesis Insights , 2004, Science.
[5] Rafael A Irizarry,et al. Exploration, normalization, and genotype calls of high-density oligonucleotide SNP array data. , 2006, Biostatistics.
[6] F. Gotch,et al. T‐cell re‐population in HIV‐infected children on highly active anti‐retroviral therapy (HAART) , 2001, Clinical and experimental immunology.
[7] J. Aster,et al. Leukemia-Associated Mutations within the NOTCH1 Heterodimerization Domain Fall into at Least Two Distinct Mechanistic Classes , 2006, Molecular and Cellular Biology.
[8] Christof von Kalle,et al. and insertional genotoxicity Cell culture assays reveal the importance of retroviral vector design for , 2006 .
[9] Tactggtat Agcctagatgtgtttaga,et al. J-mediated Translocations in Lymphoid Neoplasms : A Functional Assessment of Genomic Instability by Cryptic Sites , 2001 .
[10] Zhiyuan Luo,et al. Prospective gene expression analysis accurately subtypes acute leukaemia in children and establishes a commonality between hyperdiploidy and t(12;21) in acute lymphoblastic leukaemia , 2005, British journal of haematology.
[11] T. Lu,et al. Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. , 2006, Proceedings of the National Academy of Sciences of the United States of America.
[12] Clelia Di Serio,et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration , 2006, Nature Biotechnology.
[13] N. Tanaka,et al. Cloning of the gamma chain of the human IL-2 receptor. , 1992, Science.
[14] Jean YH Yang,et al. Bioconductor: open software development for computational biology and bioinformatics , 2004, Genome Biology.
[15] T. Rabbitts,et al. The role of LMO2 in development and in T cell leukemia after chromosomal translocation or retroviral insertion. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[16] Alessandro Aiuti,et al. Hot spots of retroviral integration in human CD34+ hematopoietic cells. , 2007, Blood.
[17] David A. Williams,et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[18] F. Deist,et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. , 2000, Science.
[19] Kathryn L. Parsley,et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[20] M. Eguchi,et al. NOTCH1 mutation can be an early, prenatal genetic event in T-ALL. , 2008, Blood.
[21] Christine Kinnon,et al. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. , 2007, The Journal of clinical investigation.
[22] Christof von Kalle,et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. , 2003, The New England journal of medicine.
[23] Yang Du,et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 , 2006, Nature Medicine.
[24] L. Foroni,et al. Notch-1 Mutations Are Secondary Events in Some Patients with T-Cell Acute Lymphoblastic Leukemia , 2007, Clinical Cancer Research.
[25] Shawn M. Burgess,et al. Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration , 2003, Science.
[26] M. Reinders,et al. Ectopic retroviral expression of LMO2, but not IL2Rγ, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy , 2007, Leukemia.
[27] Dick de Ridder,et al. New insights on human T cell development by quantitative T cell receptor gene rearrangement studies and gene expression profiling , 2005, The Journal of experimental medicine.
[28] Christine Kinnon,et al. Mutations in TNFRSF13B Encoding TACI Are Associated With Common Variable Immunodeficiency in Humans , 2006, Pediatrics.
[29] N. Greenberg,et al. NKG2D-deficient mice are defective in tumor surveillance in models of spontaneous malignancy. , 2008, Immunity.
[30] A. Fischer,et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. , 2002, The New England journal of medicine.
[31] J. Barata,et al. Interleukin-7 in T-cell acute lymphoblastic leukemia: An extrinsic factor supporting leukemogenesis? , 2005, Leukemia & lymphoma.
[32] A. Schambach,et al. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells , 2007, Blood.
[33] Hanno Glimm,et al. High-resolution insertion-site analysis by linear amplification–mediated PCR (LAM-PCR) , 2007, Nature Methods.
[34] F. Alt,et al. Activating Notch1 mutations in mouse models of T-ALL. , 2005, Blood.
[35] T. Rabbitts,et al. The oncogenic LIM protein Rbtn2 causes thymic developmental aberrations that precede malignancy in transgenic mice. , 1995, Oncogene.
[36] Bruce Aronow,et al. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. , 2007, The Journal of clinical investigation.
[37] F. Bushman,et al. Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences , 2004, PLoS biology.
[38] A. Mortellaro,et al. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning , 2002, Science.
[39] A. Look,et al. Notch 1 activation in the molecular pathogenesis of T-cell acute lymphoblastic leukaemia , 2006, Nature Reviews Cancer.