Alpharetroviral self-inactivating vectors: long-term transgene expression in murine hematopoietic cells and low genotoxicity.

[1]  Sven Diederichs,et al.  The hallmarks of cancer , 2012, RNA biology.

[2]  A. Schambach,et al.  Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells. , 2011, Blood.

[3]  Donald B Kohn,et al.  Update on gene therapy for immunodeficiencies. , 2010, Clinical immunology.

[4]  A. Schambach,et al.  Self-Inactivating Alpharetroviral Vectors with a Split-Packaging Design , 2010, Journal of Virology.

[5]  Hans Martin,et al.  Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease , 2010, Nature Medicine.

[6]  Luca Biasco,et al.  Comprehensive genomic access to vector integration in clinical gene therapy , 2009, Nature Medicine.

[7]  A. Schambach,et al.  Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[8]  G Opelz,et al.  QuickMap: a public tool for large-scale gene therapy vector insertion site mapping and analysis , 2009, Gene Therapy.

[9]  Christof von Kalle,et al.  The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. , 2009, The Journal of clinical investigation.

[10]  Mason A. Israel,et al.  Lin−Sca1+Kit− Bone Marrow Cells Contain Early Lymphoid-Committed Precursors That Are Distinct from Common Lymphoid Progenitors1 , 2008, The Journal of Immunology.

[11]  Christine Kinnon,et al.  Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. , 2008, The Journal of clinical investigation.

[12]  F. Bushman,et al.  Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. , 2008, The Journal of clinical investigation.

[13]  T. Wolfsberg,et al.  Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

[14]  A. Schambach,et al.  Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16 , 2008, Leukemia.

[15]  A. Schambach,et al.  Physiological promoters reduce the genotoxic risk of integrating gene vectors. , 2008, 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]  Daniel G. Miller,et al.  Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

[18]  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.

[19]  A. Schambach,et al.  Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors , 2006, Gene Therapy.

[20]  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.

[21]  A. Schambach,et al.  Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression , 2006, Gene Therapy.

[22]  David A. Williams,et al.  Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[23]  H. Lodish,et al.  Angiopoietin-like proteins stimulate ex vivo expansion of hematopoietic stem cells , 2006, Nature Medicine.

[24]  N. Copeland,et al.  Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. , 2005, Blood.

[25]  J. Ellis Silencing and variegation of gammaretrovirus and lentivirus vectors. , 2005, Human gene therapy.

[26]  Paul Shinn,et al.  Integration Targeting by Avian Sarcoma-Leukosis Virus and Human Immunodeficiency Virus in the Chicken Genome , 2005, Journal of Virology.

[27]  H. Lodish,et al.  Murine hematopoietic stem cells change their surface phenotype during ex vivo expansion. , 2005, Blood.

[28]  J. Coffin,et al.  Relationship between retroviral DNA-integration-site selection and host cell transcription. , 2005, Proceedings of the National Academy of Sciences of the United States of America.

[29]  J. Itskovitz‐Eldor,et al.  CD133-positive hematopoietic stem cell "stemness" genes contain many genes mutated or abnormally expressed in leukemia. , 2005, Stem cells.

[30]  Christof von Kalle,et al.  Distinct Genomic Integration of MLV and SIV Vectors in Primate Hematopoietic Stem and Progenitor Cells , 2004, PLoS biology.

[31]  R. Stoyanova,et al.  Genome-Wide Analyses of Avian Sarcoma Virus Integration Sites , 2004, Journal of Virology.

[32]  F. Bushman,et al.  Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences , 2004, PLoS biology.

[33]  J. Shipman,et al.  Design and synthesis of 3,7-diarylimidazopyridines as inhibitors of the VEGF-receptor KDR. , 2004, Bioorganic & medicinal chemistry letters.

[34]  Shawn M. Burgess,et al.  Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration , 2003, Science.

[35]  C. von Kalle,et al.  Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. , 2003, Blood.

[36]  Paul Shinn,et al.  HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots , 2002, Cell.

[37]  P. Zipfel,et al.  In Vivo Analysis of Retroviral Enhancer Mutations in Hematopoietic Cells: SP1/EGR1 and ETS/GATA Motifs Contribute to Long Terminal Repeat Specificity , 2002, Journal of Virology.

[38]  S. Jacobsen,et al.  Upregulation of Flt3 expression within the bone marrow Lin(-)Sca1(+)c-kit(+) stem cell compartment is accompanied by loss of self-renewal capacity. , 2001, Immunity.

[39]  M. Pfaffl,et al.  A new mathematical model for relative quantification in real-time RT-PCR. , 2001, Nucleic acids research.

[40]  C. von Kalle,et al.  Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. , 2001, Human gene therapy.

[41]  J. Weidhaas,et al.  Relationship between Retroviral DNA Integration and Gene Expression , 2000, Journal of Virology.

[42]  D. Trono,et al.  A Third-Generation Lentivirus Vector with a Conditional Packaging System , 1998, Journal of Virology.

[43]  F. Cosset,et al.  Inverse targeting of retroviral vectors: selective gene transfer in a mixed population of hematopoietic and nonhematopoietic cells. , 1998, Blood.

[44]  Hiromitsu Nakauchi,et al.  Long-Term Lymphohematopoietic Reconstitution by a Single CD34-Low/Negative Hematopoietic Stem Cell , 1996, Science.

[45]  H. Nakauchi,et al.  In vivo self-renewal of c-Kit+ Sca-1+ Lin(low/-) hemopoietic stem cells. , 1996, Journal of immunology.

[46]  D. Kohn,et al.  Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. , 1994, Proceedings of the National Academy of Sciences of the United States of America.

[47]  B. Fehse,et al.  Retroviral insertion site analysis in dominant haematopoietic clones. , 2009, Methods in molecular biology.

[48]  Christof von Kalle,et al.  and insertional genotoxicity Cell culture assays reveal the importance of retroviral vector design for , 2006 .

[49]  L. Zentilin,et al.  Variegation of retroviral vector gene expression in myeloid cells , 2000, Gene Therapy.