List of works by Manfred Schmidt

A Graph Based Framework to Model Virus Integration Sites

scientific article published on 30 November 2015

A Hybrid Vector for Ligand-Directed Tumor Targeting and Molecular Imaging

scientific article published on 01 April 2006

A largely random AAV integration profile after LPLD gene therapy

scientific article published on 16 June 2013

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

scientific article published in January 2003

AAVvector-mediated in vivo reprogramming into pluripotency

scientific article published in Nature Communications

Adeno-associated Vector Toxicity-To Be or Not to Be?

scientific article published on November 2015

Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle

scientific article published on 4 June 2008

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

scientific article

An unbiased genome-wide analysis of zinc-finger nuclease specificity

scientific article

Analysis of the clonal repertoire of gene-corrected cells in gene therapy

scientific article published in January 2012

Analyzing the number of common integration sites of viral vectors--new methods and computer programs

scientific article

Biodistribution of LV-TSTA transduced rat bone marrow cells used for "ex-vivo" regional gene therapy for bone repair

scientific article published on 12 August 2015

Bioinformatic clonality analysis of next-generation sequencing-derived viral vector integration sites

scientific article

Biosafety considerations using gamma-retroviral vectors in gene therapy

scientific article published on December 2013

Cell cycle status of CD34(+) hemopoietic stem cells determines lentiviral integration in actively transcribed and development-related genes

scientific article

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

scientific article published on 6 July 2006

Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

scientific article published on January 2004

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial

scientific article published on 7 December 2004

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

scientific article published on 17 March 2003

Common integration sites of published datasets identified using a graph-based framework

scientific article published on 29 November 2015

Comparative clonal analysis of reconstitution kinetics after transplantation of hematopoietic stem cells gene marked with a lentiviral SIN or a γ-retroviral LTR vector

scientific article

Comparative next-generation sequencing of adeno-associated virus inverted terminal repeats.

scientific article published on May 2014

Comparison Between Several Integrase-defective Lentiviral Vectors Reveals Increased Integration of an HIV Vector Bearing a D167H Mutant

scientific article

Comparison of lentiviral and sleeping beauty mediated αβ T cell receptor gene transfer

scientific article

Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells.

scientific article published on April 2003

Comprehensive genomic access to vector integration in clinical gene therapy

scientific article published on 22 November 2009

Comprehensive genomic characterization of gene therapy-induced T-cell acute lymphoblastic leukemia

scientific article published on 03 March 2020

Control of chronic lymphocytic leukemia development by clonally-expanded CD8+ T-cells that undergo functional exhaustion in secondary lymphoid tissues

scientific article published on 28 September 2018

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

scientific article

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

scientific article

Corrigendum to "Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice".

scientific article published on 3 November 2005

Detection of retroviral integration sites by linear amplification-mediated PCR and tracking of individual integration clones in different samples

scientific article

Dissecting the genomic complexity underlying medulloblastoma

scientific article

Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells

scientific article

Distinct types of tumor-initiating cells form human colon cancer tumors and metastases

scientific article published on 01 October 2011

Effective gene therapy with nonintegrating lentiviral vectors

scientific article

Efficient Nuclease-Directed Integration of Lentivirus Vectors into the Human Ribosomal DNA Locus

scientific article published on 23 May 2020

Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing.

scientific article published in May 2003

Efficient marking of human cells with rapid but transient repopulating activity in autografted recipients

scientific article published on 21 April 2005

Engineered dendritic cells from cord blood and adult blood accelerate effector T cell immune reconstitution against HCMV

scientific article published on 7 January 2015

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

scientific article published on 27 October 2015

Evidence of similar effects of short-term culture on the initial repopulating activity of mobilized peripheral blood transplants assessed in NOD/SCID-beta2microglobulin(null) mice and in autografted patients

scientific article published on 01 January 2005

Extensive methylation of promoter sequences silences lentiviral transgene expression during stem cell differentiation in vivo

scientific article

Fanconi anemia gene editing by the CRISPR/Cas9 system

scientific article

From bench to bedside: preclinical evaluation of a self-inactivating gammaretroviral vector for the gene therapy of X-linked chronic granulomatous disease.

scientific article published on June 2013

GENE-IS: Time-Efficient and Accurate Analysis of Viral Integration Events in Large-Scale Gene Therapy Data

scientific article

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

scientific article published in August 2007

Gene therapy for Fanconi anemia: one step closer to the clinic.

scientific article published on February 2012

Gene therapy for Wiskott-Aldrich Syndrome-Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis

scientific article published on 30 October 2014

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

scientific article

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

scientific article

Gene therapy: therapeutic gene causing lymphoma

scientific article (publication date: 27 April 2006)

Generation of lentivirus-induced dendritic cells under GMP-compliant conditions for adaptive immune reconstitution against cytomegalovirus after stem cell transplantation

scientific article published on 22 July 2015

Genetic subclone architecture of tumor clone-initiating cells in colorectal cancer.

scientific article published in June 2017

Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification

scientific article published on 12 February 2017

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing ⬇️

scientific article published on July 8, 2010

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

scientific article

Hematopoietic activity of human short-term repopulating cells in mobilized peripheral blood cell transplants is restricted to the first 5 months after transplantation.

scientific article published on 9 April 2010

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

scientific article

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

scientific article (publication date: June 2006)

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

scientific article

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

scientific article

High-resolution analysis of the human T-cell receptor repertoire

scientific article published in September 2015

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

scientific article published in December 2007

High-throughput monitoring of integration site clonality in preclinical and clinical gene therapy studies

scientific article published on April 2015

Hot spots of retroviral integration in human CD34+ hematopoietic cells

scientific article

Identification of NY-BR-1-specific CD4(+) T cell epitopes using HLA-transgenic mice

scientific article

Identity, potency, in vivo viability, and scaling up production of lentiviral vector-induced dendritic cells for melanoma immunotherapy

scientific article

Impact of raltegravir on HIV-1 RNA and DNA forms following initiation of antiretroviral therapy in treatment-naive patients

scientific article published on 23 June 2014

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

scientific article published on 12 August 2019

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model.

scientific article published on 24 April 2017

Induced pluripotent mesenchymal stromal cell clones retain donor-derived differences in DNA methylation profiles.

scientific article

Insertion site pattern: global approach by linear amplification-mediated PCR and mass sequencing

scientific article published in January 2012

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

scientific article published on 23 August 2011

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

scientific article published on September 2008

Integrase-deficient lentiviral vectors mediate efficient gene transfer to human vascular smooth muscle cells with minimal genotoxic risk

scientific article

Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

scientific article

Integration of retroviral vectors

scientific article published on 14 September 2012

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

scientific article

Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice

scientific article published on 14 August 2013

Integrative genomic analyses reveal an androgen-driven somatic alteration landscape in early-onset prostate cancer

scientific article

Interaction of vectors and parental viruses with the host genome

scientific article

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

scientific article published on 25 May 2016

Is IL2RG oncogenic in T-cell development?: X-SCID transgene leukaemogenicity (reply)

scientific article published in Nature

Lentiviral Vector Promoter is Decisive for Aberrant Transcript Formation

scientific article published on 21 August 2017

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

scientific article published in January 2012

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

scientific article

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

scientific article

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

scientific article published on 14 March 2011

Lentiviral vector integration profiles differ in rodent postmitotic tissues

scientific article

Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis

scientific article published on 17 October 2002

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer

scientific article published on 13 January 2013

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs.

scientific article published on 29 September 2015

Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors

scientific article

Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis.

scientific article

Linear amplification mediated PCR--localization of genetic elements and characterization of unknown flanking DNA.

scientific article

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

scientific article

Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia

scientific article published in March 2004

Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors.

scientific article

Mapping Active Gene-Regulatory Regions in Human Repopulating Long-Term HSCs

article

Mapping the precision of genome editing.

scientific article published in February 2015

Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model

scientific article published on November 2003

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

scientific article published on November 2002

Murine leukemia induced by retroviral gene marking

scientific article published on 01 April 2002

Next-generation sequencing of cancer consensus genes in lymphoma

scientific article published on 14 June 2013

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

scientific article

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

scientific article published in September 2013

Patient-derived xenografts of gastrointestinal cancers are susceptible to rapid and delayed B-lymphoproliferation

scientific article

Pharmacologically regulated in vivo selection in a large animal

scientific article published in September 2002

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

scientific article published on 17 May 2016

Phenotypic differentiation does not affect tumorigenicity of primary human colon cancer initiating cells.

scientific article published on 8 December 2015

Polyclonal long-term repopulating stem cell clones in a primate model

scientific article published in October 2002

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

scientific article

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

scientific article

Presence of a trs-Like Motif Promotes Rep-Mediated Wild-Type Adeno-Associated Virus Type 2 Integration.

scientific article published on 13 May 2015

Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

scientific article published on 24 December 2019

Real-time definition of non-randomness in the distribution of genomic events

scientific article

Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

scientific article published on 07 March 2016

Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells ⬇️

scientific article published on 2 June 2005

Reply to "Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV"

scientific article published on April 2016

Reply to: NGS library preparation may generate artifactual integration sites of AAV vectors.

scientific article published in June 2014

Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial

scientific article published on 30 August 2011

Retroviral vectors: post entry events and genomic alterations

scientific article

Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria

scientific article published on December 2013

Selective inhibition of tumor growth by clonal NK cells expressing an ErbB2/HER2-specific chimeric antigen receptor

scientific article

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells

scientific article

Short-term repopulating cells with myeloid potential in human mobilized peripheral blood do not have a side population (SP) phenotype

scientific article published on 30 May 2006

Sleeping beauty transposition from nonintegrating lentivirus

scientific article

Spatial-Temporal Variations in Atmospheric Factors Contribute to SARS-CoV-2 Outbreak

scientific article published on 27 May 2020

Spatial-temporal variations of atmospheric factors contribute to SARS-CoV-2 outbreak

scientific article published on 01 May 2020

Spatially clustered loci with multiple enhancers are frequent targets of HIV-1 integration

scientific article published on 06 September 2019

Stable differentiation and clonality of murine long-term hematopoiesis after extended reduced-intensity selection for MGMT P140K transgene expression

scientific article

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques

scientific article published on 31 May 2011

Stable long-term blood formation by stem cells in murine steady-state hematopoiesis

scientific article published in September 2012

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

scientific article

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

scientific article

Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing

scientific article published on 15 May 2015

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

scientific article published on 09 September 2019

Succession of transiently active tumor-initiating cell clones in human pancreatic cancer xenografts

scientific article

Systematic comparative study of computational methods for T-cell receptor sequencing data analysis

scientific article

TALEN-based gene correction for epidermolysis bullosa.

scientific article published on 2 April 2013

TCR sequences and tissue distribution discriminate the subsets of naïve and activated/memory Treg cells in mice

scientific article

Targeted resequencing for analysis of clonal composition of recurrent gene mutations in chronic lymphocytic leukaemia

scientific article

The Clonal Fate of Live Cells

scientific article

The Conjugative Relaxase TrwC Promotes Integration of Foreign DNA in the Human Genome

scientific article published on 14 April 2017

The Contained Self-Reactive Peripheral T Cell Repertoire: Size, Diversity, and Cellular Composition

scientific article published on 20 July 2015

The LAM-PCR Method to Sequence LV Integration Sites

scientific article published on January 2016

The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis

scientific article published on 13 November 2012

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

scientific article published on 23 March 2009

The inherent differentiation program of short-term hematopoietic repopulating cells changes during human ontogeny

scientific article published in May 2010

The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity

scientific article

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques

scientific article published on 05 December 2018

Thymus-autonomous T cell development in the absence of progenitor import

scientific article

Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory.

scientific article

Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease

scientific article

Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors

scientific article published in August 2009

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

scientific article

VSeq-Toolkit: Comprehensive Computational Analysis of Viral Vectors in Gene Therapy

scientific article published on 30 March 2020

Vector integration and tumorigenesis

scientific article

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

scientific article published in August 2007

Paulina is supported by:

About Paulina

Help