List of works by Axel Schambach

A Lentiviral Fluorescent Genetic Barcoding System for Flow Cytometry-Based Multiplex Tracking

scientific article

A differentiation checkpoint limits hematopoietic stem cell self-renewal in response to DNA damage

scientific journal article

A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells

scientific article

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

scientific article

A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice

scientific article

A suicide gene approach using the human pro-apoptotic protein tBid inhibits HIV-1 replication

scientific article

A tumor-derived population (SCCOHT-1) as cellular model for a small cell ovarian carcinoma of the hypercalcemic type

scientific article published on 8 May 2012

Advantages and applications of CAR-expressing natural killer cells

scientific article

All-in-One inducible lentiviral vector systems based on drug controlled FLP recombinase

scientific article published on 14 February 2014

Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes

scientific article published on 26 April 2016

Alpharetroviral vector-mediated gene therapy for X-CGD: functional correction and lack of aberrant splicing

scientific article published on 4 December 2012

Alpharetroviral vectors: from a cancer-causing agent to a useful tool for human gene therapy

scientific article published on 05 December 2014

An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries

scientific article

Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery

scientific article

Biosafety challenges for use of lentiviral vectors in gene therapy

scientific article published on December 2013

Biosafety features of lentiviral vectors

scientific article published on February 2013

Bromo- and extraterminal domain chromatin regulators serve as cofactors for murine leukemia virus integration

scientific article published on 18 September 2013

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

scientific article published on 6 July 2006

Cellular restriction of retrovirus particle-mediated mRNA transfer

scientific article

Chemoprotection of murine hematopoietic cells by combined gene transfer of cytidine deaminase (CDD) and multidrug resistance 1 gene (MDR1).

scientific article published on 12 December 2015

Clinical application of lentiviral vectors - concepts and practice

scientific article published on December 2008

Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model

scientific article published on 7 October 2014

Comparison of Different Cytokine Conditions Reveals Resveratrol as a New Molecule for Ex Vivo Cultivation of Cord Blood-Derived Hematopoietic Stem Cells

scientific article

Comparison of the activity and pluripotency maintaining potential of human leukemia inhibitory factor (LIF) produced in E.coli and CHO cells

scientific article

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

scientific article

Deciphering the impact of parameters influencing transgene expression kinetics after repeated cell transduction with integration-deficient retroviral vectors

scientific article published on 27 February 2015

Design and production of retro- and lentiviral vectors for gene expression in hematopoietic cells

scientific article

Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy

scientific article

Dose response and clonal variability of lentiviral tetracycline-regulated vectors in murine hematopoietic cells

scientific article published on 24 March 2014

Efficient generation of gene-modified human natural killer cells via alpharetroviral vectors

scientific article published on 25 August 2015

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy

scientific article published on 17 May 2017

Evaluating a ligation-mediated PCR and pyrosequencing method for the detection of clonal contribution in polyclonal retrovirally transduced samples

scientific article published on 14 March 2013

Expanding Flp-RMCE options: the potential of Recombinase Mediated Twin-Site Targeting (RMTT).

scientific article

Expression and purification of bioactive soluble murine stem cell factor from recombinant Escherichia coli using thioredoxin as fusion partner

scientific article published on 22 January 2011

Expression of the ETS transcription factor GABPα is positively correlated to the BCR-ABL1/ABL1 ratio in CML patients and affects imatinib sensitivity in vitro

scientific article

FAS-based cell depletion facilitates the selective isolation of mouse induced pluripotent stem cells

scientific article

Function and safety of lentivirus-mediated gene transfer for CSF2RA-deficiency

scientific article

GTPase domain driven dimerization of SEPT7 is dispensable for the critical role of septins in fibroblast cytokinesis

scientific article published on 28 January 2016

Gammaretroviral vectors: biology, technology and application

scientific article

Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy

scientific article published on 12 February 2016

Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia

scientific article

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

scientific article

Gene therapy: X-SCID transgene leukaemogenicity

scientific article

Generation and genetic modification of induced pluripotent stem cells

scientific article published on July 2010

Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform

scientific article

Generation of HLA-universal iPSCs-derived megakaryocytes and platelets for survival under refractoriness conditions

scientific article

Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells

scientific article

Genetic deletion of SEPT7 reveals a cell type-specific role of septins in microtubule destabilization for the completion of cytokinesis

scientific article (publication date: August 2014)

Genetic modification of lymphocytes by retrovirus-based vectors

scientific article published on 17 September 2012

Genetic reporter analysis reveals an expandable reservoir of OCT4+ cells in adult skin

scientific article

Glycomic Characterization of Induced Pluripotent Stem Cells Derived from a Patient Suffering from Phosphomannomutase 2 Congenital Disorder of Glycosylation (PMM2-CDG)

scientific article

Highly significant antiviral activity of HIV-1 LTR-specific tre-recombinase in humanized mice

scientific article published in 2013

Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase

scientific article (publication date: 2 December 2016)

Improved killing of ovarian cancer stem cells by combining a novel CAR-based immunotherapy and chemotherapy

scientific article published on 24 August 2017

Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors

scientific article published on 3 April 2007

Induced pluripotent stem cells generated from adult bone marrow-derived cells of the nonhuman primate (Callithrix jacchus) using a novel quad-cistronic and excisable lentiviral vector

scientific article published on December 2012

Induction of a mature hepatocyte phenotype in adult liver derived progenitor cells by ectopic expression of transcription factors

scientific article published on 24 February 2011

Induction of pluripotent stem cells from a cynomolgus monkey using a polycistronic simian immunodeficiency virus-based vector, differentiation toward functional cardiomyocytes, and generation of stably expressing reporter lines

scientific article

Inherited DOCK2 Deficiency in Patients with Early-Onset Invasive Infections

scientific article published on June 2015

Inhibition of miRNA-212/132 improves the reprogramming of fibroblasts into induced pluripotent stem cells by de-repressing important epigenetic remodelling factors

scientific article

Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

scientific article

Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis

scientific article published on October 2017

LEF-1 is crucial for neutrophil granulocytopoiesis and its expression is severely reduced in congenital neutropenia

scientific article

Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease

scientific article

Lentiviral Protein Transfer Vectors Are an Efficient Vaccine Platform and Induce a Strong Antigen-Specific Cytotoxic T Cell Response

scientific article published on 17 June 2015

Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia

scientific article published on 20 April 2017

Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice

scientific article published on 6 February 2015

Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemia

scientific article

Lentiviral vector system for coordinated constitutive and drug controlled tetracycline-regulated gene co-expression

scientific article

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

scientific article published on 24 April 2007

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

scientific article published on 9 September 2016

Lentivirus-induced dendritic cells for immunization against high-risk WT1(+) acute myeloid leukemia

scientific article

Long noncoding RNA Chast promotes cardiac remodeling

scientific article

Loss-of-function mutations in the IL-21 receptor gene cause a primary immunodeficiency syndrome

scientific article published on 25 February 2013

MN1 overexpression induces acute myeloid leukemia in mice and predicts ATRA resistance in patients with AML.

scientific article

Massive Clonal Selection and Transiently Contributing Clones During Expansion of Mesenchymal Stem Cell Cultures Revealed by Lentiviral RGB-Barcode Technology

scientific article published on 31 March 2016

Mesenchymal stem cells directly interact with breast cancer cells and promote tumor cell growth in vitro and in vivo

scientific article published on 9 September 2013

Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia

scientific article

Minicircle performance depending on S/MAR-nuclear matrix interactions

scientific article

Modified lentiviral LTRs allow Flp recombinase-mediated cassette exchange and in vivo tracing of "factor-free" induced pluripotent stem cells

scientific article

Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration

scientific article

Multiplexing RMCE: versatile extensions of the Flp-recombinase-mediated cassette-exchange technology

scientific article

Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency

scientific article published on 20 July 2016

NAMPT is essential for the G-CSF-induced myeloid differentiation via a NAD(+)-sirtuin-1-dependent pathway

scientific article

New way of regulating alternative splicing in retroviruses: the promoter makes a difference

scientific article published on 17 January 2007

Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery

scientific article

Novel self-inactivating vectors for reconstitution of Wiskott-Aldrich syndrome

scientific article

Optimization of human NK cell manufacturing: Fully-automated separation, improved <i>ex vivo </i>expansion using IL-21 with autologous feeder cells and generation of anti-CD123-CAR-expressing effector cells

scientific article

Overcoming reprogramming resistance of Fanconi anemia cells

scientific article

Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency

scientific article published on 19 December 2014

Physiological promoters reduce the genotoxic risk of integrating gene vectors

scientific article

Potent and reversible lentiviral vector restriction in murine induced pluripotent stem cells

scientific article published on 31 May 2017

Preclinical validation: LV/IL-12 transduction of patient leukemia cells for immunotherapy of AML.

scientific article published on 7 December 2016

Preparation of bioactive soluble human leukemia inhibitory factor from recombinant Escherichia coli using thioredoxin as fusion partner

scientific article published on 8 April 2010

Production and purification of TGFb-1 in CHO-Cells

scientific article published on 16 December 2011

Protein transduction from retroviral Gag precursors

scientific article

Pseudotype-independent nonspecific uptake of gammaretroviral and lentiviral particles in human cells

scientific article

Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells

scientific article

Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin

scientific article

Reticular dysgenesis-associated AK2 protects hematopoietic stem and progenitor cell development from oxidative stress

scientific article published on 6 July 2015

Retroviral Vectors for Cancer Gene Therapy

scientific article published on January 2016

Retroviral and Transposon-Based Tet-Regulated All-In-One Vectors with Reduced Background Expression and Improved Dynamic Range ⬇️

scientific article published on December 19, 2010

Retroviral protein transfer: falling apart to make an impact

scientific article published on October 2012

Retrovirus vectors: toward the plentivirus?

scientific article

Retrovirus-based mRNA transfer for transient cell manipulation

scientific article

Retrovirus-based vectors for transient and permanent cell modification

scientific article published on 30 September 2015

Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency

scientific article

Safety of gene therapy: new insights to a puzzling case

scientific article published on January 2014

Scavenger receptor class B member 1 (SCARB1) variants modulate hepatitis C virus replication cycle and viral load

scientific article published on 28 March 2017

Self-inactivating alpharetroviral vectors with a split-packaging design

scientific article published on 21 April 2010

Skin tissue generation by laser cell printing

scientific article

State-of-the-art lentiviral vectors for research use: risk assessment and biosafety recommendations

scientific article published on December 2009

Stem cell attraction via SDF-1α expressing fat tissue grafts

scientific article published on 22 December 2012

Stem cell marking with promotor-deprived self-inactivating retroviral vectors does not lead to induced clonal imbalance

scientific article

Sustained knockdown of a disease-causing gene in patient-specific induced pluripotent stem cells using lentiviral vector-based gene therapy

scientific article published on 07 August 2013

TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells

scientific article published on 3 August 2015

The CpG-sites of the CBX3 ubiquitous chromatin opening element are critical structural determinants for the anti-silencing function

scientific article published on 11 August 2017

The heteromeric transcription factor GABP activates the ITGAM/CD11b promoter and induces myeloid differentiation

scientific article published on 10 July 2015

The phenotype of human STK4 deficiency

scientific article published on 31 January 2012

The role of induced pluripotent stem cells in research and therapy of primary immunodeficiencies

scientific article published on 25 July 2012

Toward a safer integration profile of MLV-based retroviral vectors

scientific article

Tumor cells escape suicide gene therapy by genetic and epigenetic instability

scientific article published on 12 August 2004

Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells

scientific article

Viral and Synthetic RNA Vector Technologies and Applications

scientific article

Viral and non-viral approaches for transient delivery of mRNA and proteins

scientific article published on October 2011

miR-155 is associated with the leukemogenic potential of the class IV granulocyte colony-stimulating factor receptor in CD34⁺ progenitor cells

scientific article published on January 2014

miR-99a/100~125b tricistrons regulate hematopoietic stem and progenitor cell homeostasis by shifting the balance between TGFβ and Wnt signaling

scientific article published on April 2014

miRNA screening reveals a new miRNA family stimulating iPS cell generation via regulation of Meox2.

scientific article

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