The β-globin Replicator greatly enhances the potential of S/MAR based episomal vectors for gene transfer into human haematopoietic progenitor cells

[1]  M. Essand,et al.  Safe engineering of CAR T cells for adoptive cell therapy of cancer using long‐term episomal gene transfer , 2016, EMBO molecular medicine.

[2]  P. Stephens,et al.  Chromatin Function Modifying Elements in an Industrial Antibody Production Platform - Comparison of UCOE, MAR, STAR and cHS4 Elements , 2015, PloS one.

[3]  Rafael J. Yáñez-Muñoz,et al.  Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors. , 2014, Human gene therapy.

[4]  H. Lipps,et al.  S/MAR sequence confers long-term mitotic stability on non-integrating lentiviral vector episomes without selection , 2014, Nucleic acids research.

[5]  N. Shimizu,et al.  Dissection of the Beta-Globin Replication-Initiation Region Reveals Specific Requirements for Replicator Elements during Gene Amplification , 2013, PloS one.

[6]  H. Lipps,et al.  Genomic cis-acting Sequences Improve Expression and Establishment of a Nonviral Vector , 2013, Molecular therapy. Nucleic acids.

[7]  A. Baiker,et al.  A Novel Adenoviral Hybrid-vector System Carrying a Plasmid Replicon for Safe and Efficient Cell and Gene Therapeutic Applications , 2013, Molecular therapy. Nucleic acids.

[8]  Petter Holme,et al.  The Effect of Disease-Induced Mortality on Structural Network Properties , 2013, PloS one.

[9]  D. Odom,et al.  CTCF and Cohesin: Linking Gene Regulatory Elements with Their Targets , 2013, Cell.

[10]  G. Romano Development of Safer Gene Delivery Systems to Minimize the Risk of Insertional Mutagenesis-Related Malignancies: A Critical Issue for the Field of Gene Therapy , 2012, ISRN oncology.

[11]  A. Baiker,et al.  A colony-forming assay for determining the establishment efficiency of S/MAR-containing nonviral episomal expression vectors. , 2012, Cold Spring Harbor protocols.

[12]  A. Ensser,et al.  The Insulator Protein CTCF Binding Sites in the orf73/LANA Promoter Region of Herpesvirus Saimiri Are Involved in Conferring Episomal Stability in Latently Infected Human T Cells , 2011, Journal of Virology.

[13]  D. Scherman,et al.  pFARs, Plasmids free of antibiotic resistance markers, display high‐level transgene expression in muscle, skin and tumour cells , 2010, The journal of gene medicine.

[14]  A. Baiker,et al.  pEPito: a significantly improved non-viral episomal expression vector for mammalian cells , 2010, BMC biotechnology.

[15]  A. Schambach,et al.  Minicircle performance depending on S/MAR-nuclear matrix interactions. , 2010, Journal of molecular biology.

[16]  A. Sgourou,et al.  Physiological levels of HBB transgene expression from S/MAR element-based replicating episomal vectors. , 2009, Journal of biotechnology.

[17]  A. Thrasher,et al.  The functional role of S/MARs in episomal vectors as defined by the stress-induced destabilization profile of the vector sequences. , 2009, Journal of molecular biology.

[18]  A. Miller,et al.  Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector , 2008, Gene Therapy.

[19]  T. Cremer,et al.  Establishment and mitotic stability of an extra-chromosomal mammalian replicon , 2007, BMC Cell Biology.

[20]  M. Papa,et al.  Genetically modified pigs produced with a nonviral episomal vector , 2006, Proceedings of the National Academy of Sciences.

[21]  M. Aladjem,et al.  Cooperative sequence modules determine replication initiation sites at the human beta-globin locus. , 2006, Human molecular genetics.

[22]  Chengpeng Bi,et al.  Correlations between Scaffold/matrix Attachment Region (s/mar) Binding Activity and Dna Duplex Destabilization Energy , 2022 .

[23]  E. Papapetrou,et al.  Genetic modification of hematopoietic stem cells with nonviral systems: past progress and future prospects , 2005, Gene Therapy.

[24]  H. Lipps,et al.  Towards safe, non-viral therapeutic gene expression in humans , 2005, Nature Reviews Genetics.

[25]  M. Aladjem The mammalian beta globin origin of DNA replication. , 2004, Frontiers in bioscience : a journal and virtual library.

[26]  A. Baiker,et al.  Nuclear scaffold/matrix attached region modules linked to a transcription unit are sufficient for replication and maintenance of a mammalian episome. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[27]  Chengpeng Bi,et al.  WebSIDD: server for predicting stress-induced duplex destabilized (SIDD) sites in superhelical DNA. , 2004, Bioinformatics.

[28]  H. Lipps,et al.  An episomal mammalian replicon: sequence‐independent binding of the origin recognition complex , 2004, The EMBO journal.

[29]  Kathryn L. Parsley,et al.  High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter , 2002 .

[30]  F. Jönsson,et al.  An episomally replicating vector binds to the nuclear matrix protein SAF‐A in vivo , 2002, EMBO reports.

[31]  S. Raguz,et al.  LCR-mediated, long-term tissue-specific gene expression within replicating episomal plasmid and cosmid vectors , 2002, Gene Therapy.

[32]  P. Charneau,et al.  Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[33]  A. Baiker,et al.  Mitotic stability of an episomal vector containing a human scaffold/matrix-attached region is provided by association with nuclear matrix , 2000, Nature Cell Biology.

[34]  D. Schübeler,et al.  Scaffold/matrix-attached regions act upon transcription in a context-dependent manner. , 1996, Biochemistry.

[35]  A. Fire,et al.  © The American Society of Gene & Cell Therapy original article Minicircle DNA Vectors Achieve Sustained Expression Reflected by Active Chromatin and Transcriptional , 2022 .

[36]  A. Baiker,et al.  Episomal vectors for gene therapy. , 2008, Current gene therapy.

[37]  E. Papapetrou,et al.  Gene transfer into human hematopoietic progenitor cells with an episomal vector carrying an S/MAR element , 2006, Gene Therapy.

[38]  A. Baiker,et al.  Exploiting a minimal system to study the epigenetic control of DNA replication: the interplay between transcription and replication , 2004, Chromosome Research.

[39]  Kathryn L. Parsley,et al.  High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. , 2002, Human gene therapy.

[40]  A. Baiker,et al.  A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells. , 1999, Nucleic acids research.

[41]  T. Schlake,et al.  Scaffold/matrix-attached regions: structural properties creating transcriptionally active loci. , 1995, International review of cytology.