M. Schmidt

发表

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

C. von Kalle, S. Fröhling, R. Eils, 2017, The Journal of experimental medicine.

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

C. von Kalle, J. Prieto, H. Petry, 2013, Human gene therapy.

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

J. Soulier, J. Surrallés, R. Yañez, 2019, Nature Medicine.

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

C. von Kalle, J. Bueren, A. Galy, 2015, Current gene therapy.

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

David A. Williams, C. von Kalle, J. Wagner, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, D. Peebles, A. David, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

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

M. Choolani, J. Chan, Manfred Schmidt, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Hot spots of retroviral integration in human CD34+ hematopoietic cells.

Alessandro Aiuti, Christof von Kalle, Manfred Schmidt, 2007, Blood.

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

Clelia Di Serio, Alessandro Ambrosi, Luigi Naldini, 2006, Nature Biotechnology.

LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Cameron S. Osborne, A. Fischer, C. von Kalle, 2003, Science.

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Luca Biasco, Alessandro Aiuti, Maria Grazia Valsecchi, 2013, Science.

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

A. Jauch, E. Seifried, W. Wels, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

C. von Kalle, G. Zanetti, E. Stupka, 2013, Science.

Stem-cell gene therapy for the Wiskott-Aldrich syndrome.

Christof von Kalle, Christoph Klein, Rainer Blasczyk, 2010, The New England journal of medicine.

HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival

Y. Takeuchi, R. Fronza, S. Jobling, 2022, Gene Therapy.

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Manfred Schmidt, Philippe Leboulch, Cynthia C. Bartholomae, 2009, Science.

Dissecting the genomic complexity underlying medulloblastoma

Matthew J. Betts, Robert B. Russell, Roland Eils, 2012, Nature.

Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

Michael Rothe, Christof von Kalle, Raffaele Fronza, 2014, Science Translational Medicine.

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

W. Sessa, R. Mcdonald, R. Fronza, 2012, Human gene therapy.

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

Hans Martin, Barbara Burwinkel, Rongxi Yang, 2010, Nature Medicine.

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

Christine Kinnon, Christof von Kalle, Michael Hubank, 2008, The Journal of clinical investigation.

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

C. von Kalle, Y. Chérel, F. Rolling, 2008, Journal of Virology.

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

Yang Du, Manuel Grez, Hanno Glimm, 2006, Nature Medicine.

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

C. von Kalle, B. Fehse, Johann Meyer, 2005, Blood.

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

Christof von Kalle, Manfred Schmidt, Salima Hacein-Bey-Abina, 2003, The New England journal of medicine.

Murine Leukemia Induced by Retroviral Gene Marking

C. von Kalle, B. Fehse, Johann Meyer, 2002, Science.

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

C. Lilley, M. Weitzman, M. Ozawa, 2006, Cell.

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.

Rafael J. Yáñez-Muñoz, C. von Kalle, Manfred Schmidt, 2014, Human gene therapy.

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

G. Dickson, J. Bode, Manfred Schmidt, 2014, Human gene therapy.

Effective gene therapy with nonintegrating lentiviral vectors

S. E. Barker, Rafael J. Yáñez-Muñoz, J. Heckenlively, 2006, Nature Medicine.

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

C. von Kalle, L. Ailles, L. Naldini, 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

Stable Long‐Term Blood Formation by Stem Cells in Murine Steady‐State Hematopoiesis

C. von Kalle, H. Glimm, Manfred Schmidt, 2012, Stem cells.

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

Luca Biasco, Alessandro Aiuti, Christof von Kalle, 2011, EMBO molecular medicine.

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

C. von Kalle, I. Glauche, B. Fehse, 2013, Experimental hematology.

Hot spots of retroviral integration in human CD 34

Claudia Cattoglio, A. Thrasher, Manfred Schmidt, 2007 .

Hot spots of retroviral integration in human CD34 (cid:1) hematopoietic cells

Claudia Cattoglio, A. Thrasher, Manfred Schmidt, 2007 .

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

C. von Kalle, C. Klein, Manfred Schmidt, 2014, Rare diseases.

Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

C. von Kalle, L. Biasco, L. Naldini, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

Interleukin-10 receptor signaling promotes the maintenance of a PD-1int TCF-1+ CD8+ T cell population that sustains anti-tumor immunity.

E. Campo, D. Colomer, P. Lichter, 2021, Immunity.

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

Christof von Kalle, Alessandro Ambrosi, Luigi Naldini, 2009, The Journal of clinical investigation.

Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.

A. Schambach, T. Olson, P. Amrolia, 2021, The New England journal of medicine.

Gene therapy: Therapeutic gene causing lymphoma

I. Verma, V. Bottero, Manfred Schmidt, 2006, Nature.

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

C. von Kalle, A. Ganser, R. Blasczyk, 2015, Journal of Translational Medicine.

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

C. von Kalle, A. Ganser, B. Eiz-Vesper, 2015, Molecular therapy. Methods & clinical development.

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

Guillaume J. Filion, E. Zorita, R. Fronza, 2019, Nature Communications.

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

Guillaume J. Filion, E. Zorita, K. Vlahovicek, 2018, bioRxiv.

Interaction of vectors and parental viruses with the host genome.

Manfred Schmidt, Irene Gil-Farina, Manfred Schmidt, 2016, Current Opinion in Virology.

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

Axel Benner, Wiebke Werft, Wilko Weichert, 2016, Cancer letters.

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

M. Koch, C. von Kalle, W. Weichert, 2011, Cell stem cell.

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

C. von Kalle, C. Eaves, H. Glimm, 2005, Blood.

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

A. Fischer, F. Deist, M. Cavazzana‐Calvo, 2013 .

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

B. Aronow, M. Wissler, D. Kohn, 2003, Nature Medicine.

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

C. von Kalle, M. Wissler, J. Tisdale, 2002, Blood.

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

H. Hennies, M. Zenke, T. Šarić, 2013, Molecular Therapy.

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

C. von Kalle, M. Kriegsmann, W. Weichert, 2017, International journal of cancer.

The balance between the intronic miR-342 and its host gene Evl determines hematopoietic cell fate decision

C. von Kalle, C. Klein, H. Glimm, 2021, Leukemia.

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

T. Holland-Letz, C. von Kalle, C. Klein, 2018, Cell stem cell.

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

C. von Kalle, F. Tuorto, H. Glimm, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Bruce Aronow, Christof von Kalle, Jean Weissenbach, 2007, The Journal of clinical investigation.

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

H. Glimm, Manfred Schmidt, C. Kalle, 2012, Methods in enzymology.

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

Stephan Wolf, Wei Wang, Richard Gabriel, 2010, Nature Protocols.

Comprehensive genomic access to vector integration in clinical gene therapy

Luca Biasco, Alessandro Aiuti, Richard Gabriel, 2009, Nature Medicine.

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

Hanno Glimm, Manfred Schmidt, Kerstin Schwarzwaelder, 2007, Nature Methods.

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

C. von Kalle, H. Glimm, Manfred Schmidt, 2009, Methods in molecular biology.

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

V. Beneš, C. von Kalle, R. Eils, 2013, Cancer cell.

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

C. von Kalle, Victor Neduva, C. Di Serio, 2016, Cell stem cell.

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

C. von Kalle, C. Eaves, H. Glimm, 2010, Stem cells and development.

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

A. Schambach, Manfred Schmidt, U. Modlich, 2008 .

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

Christof von Kalle, Christopher Baum, Ute Modlich, 2006 .

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

C. von Kalle, M. Laukkanen, C. Dunbar, 2005, Blood.

Retroviral Vectors: Post Entry Events and Genomic Alterations

C. von Kalle, H. Glimm, Manfred Schmidt, 2011, Viruses.

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

C. von Kalle, G. Tonon, L. Naldini, 2013, Nature Methods.

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

E. Campo, D. Colomer, P. Lichter, 2018, Leukemia.

Next-generation sequencing of cancer consensus genes in lymphoma

C. von Kalle, A. Jauch, H. Glimm, 2013, Leukemia & lymphoma.

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

C. von Kalle, A. Schambach, Claudia Cattoglio, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

The landscape of genomic alterations across childhood cancers

Michael C. Heinold, Jules N. A. Kerssemakers, David C. Jones, 2018, Nature.

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

C. von Kalle, S. Karlsson, D. Trono, 2003, Blood.

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

Jeffrey C. Miller, P. Gregory, L. Naldini, 2011, Nature Biotechnology.

AAV vector-mediated in vivo reprogramming into pluripotency

D. Grimm, M. Serrano, R. Fronza, 2018, Nature Communications.

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

C. von Kalle, G. Milligan, M. Houslay, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Christine Kinnon, Christof von Kalle, Dick de Ridder, 2007, The Journal of clinical investigation.

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

C. von Kalle, H. Glimm, F. Wenz, 2015, Molecular therapy. Methods & clinical development.

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

Kathryn L. Parsley, R. Levinsky, R. Ali, 2004, The Lancet.

Fanconi anemia gene editing by the CRISPR/Cas9 system.

Christof von Kalle, Jakub Tolar, Richard Gabriel, 2015, Human gene therapy.

Real-Time Definition of Non-Randomness in the Distribution of Genomic Events

C. von Kalle, A. Fischer, M. Cavazzana‐Calvo, 2007, PloS one.

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

Gianluigi Zanetti, Christof von Kalle, Alessandra Biffi, 2011, Blood.

Longitudinal analyses of CLL in mice identify leukemia-related clonal changes including a Myc gain predicting poor outcome in patients

A. Jauch, P. Lichter, S. Stilgenbauer, 2021, Leukemia.

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

C. von Kalle, A. Fischer, Claudia Cattoglio, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.

A. Fischer, M. Cavazzana‐Calvo, P. Bougnères, 2012, Methods in Enzymology.

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.

J. Tisdale, Manfred Schmidt, A. Thompson, 2020, Blood advances.

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease

C. von Kalle, G. Veres, B. Ryu, 2014, Current gene therapy.

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

Christof von Kalle, Niraja Dighe, Alaine T. Ruthe, 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. Eaves, A. Cheung, S. Fucharoen, 2013, Stem Cells.

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

David A. Williams, C. von Kalle, D. Williams, 2007, Blood.

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

C. von Kalle, Manfred Schmidt, C. Bartholomae, 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Susan M. Schlenner, C. von Kalle, P. Fink, 2012, The Journal of experimental medicine.

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

C. von Kalle, A. Bemelmans, O. Delelis, 2014, Molecular therapy. Nucleic acids.

Overexpression of the anti-apoptotic protein AVEN contributes to increased malignancy in hematopoietic neoplasms

A. Jauch, M. H. Angelis, B. Schoell, 2013, Oncogene.

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

M. Choolani, J. Chan, Manfred Schmidt, 2018, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

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

J. Prieto, H. Petry, Valérie Ferreira, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

A largely random AAV integration profile after LPLD gene therapy

Stephan Wolf, Raffaele Fronza, Hanno Glimm, 2013, Nature Medicine.

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

C. von Kalle, R. Koup, M. Betts, 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Christof von Kalle, Jakub Tolar, Richard Gabriel, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

J. Tisdale, M. Gallagher, Manfred Schmidt, 2020 .

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

C. von Kalle, C. Brombin, L. Naldini, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

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

David A. Williams, C. von Kalle, K. Pollok, 2003, Human gene therapy.

Distinct Genomic Integration of MLV and SIV Vectors in Primate Hematopoietic Stem and Progenitor Cells

Christof von Kalle, Manfred Schmidt, Peiman Hematti, 2004, PLoS biology.

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

Laurence H. Jackson, S. Eaton, A. David, 2019, Scientific Reports.

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

Christof von Kalle, Jakub Tolar, Richard Gabriel, 2017, Molecular therapy. Methods & clinical development.

Mapping the precision of genome editing

Manfred Schmidt, R. Gabriel, C. Kalle, 2015, Nature Biotechnology.

Common clonal origin of conventional T cells and induced regulatory T cells in breast cancer patients

C. von Kalle, N. Halama, I. Macaulay, 2021, Nature Communications.

Efficient Characterization of Retro‐, Lenti‐, and Foamyvector‐Transduced Cell Populations by High‐Accuracy Insertion Site Sequencing

S. Karlsson, H. Glimm, M. Wissler, 2003, Annals of the New York Academy of Sciences.

High-Definition Mapping of Retroviral Integration Sites Defines the Fate of Allogeneic T Cells After Donor Lymphocyte Infusion

Christof von Kalle, Danilo Pellin, Alessandro Ambrosi, 2010, PloS one.

Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance in Mice with Low Genotoxic Risk

C. von Kalle, A. Annoni, L. Naldini, 2011, Hepatology.

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

C. von Kalle, S. Fröhling, E. Schröck, 2020, Leukemia.

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

Christof von Kalle, Raffaele Fronza, Hanno Glimm, 2015, Nature Communications.

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

L. Kiger, P. Touraine, F. Monpoux, 2022, Nature Medicine.

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

A. Jauch, A. Ho, H. Glimm, 2013, British journal of haematology.

Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.

H. Bitter, P. Gregory, Manfred Schmidt, 2021, The New England journal of medicine.

TALEN-based gene correction for epidermolysis bullosa.

Christof von Kalle, Jakub Tolar, Richard Gabriel, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

In vivo tracking of human hematopoiesis reveals patterns of clonal dynamics during early post-transplant and steady-state reconstitution phases

Samantha, Serio, Victor Neduva, 2018 .

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

Victor Neduva, E. Wit, L. Biasco, 2022 .

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

C. von Kalle, A. Nienhuis, C. A. Blau, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

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

Christof von Kalle, Raffaele Fronza, Manfred Schmidt, 2016, Molecular therapy. Nucleic acids.

Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes

F. Larcher, F. Cocchiarella, F. Mavilio, 2013, Gene Therapy.

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

C. Dehio, Manfred Schmidt, M. Llosa, 2017, Applied and Environmental Microbiology.

Sleeping beauty transposition from nonintegrating lentivirus.

C. Vink, R. McIvor, A. Thrasher, 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, H. Glimm, Manfred Schmidt, 2008, Blood.

C. von Kalle, C. Eaves, H. Glimm, 2005, Experimental hematology.

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

C. von Kalle, L. Biasco, R. Fronza, 2015, Science Translational Medicine.

Lentiviral vector integration profiles differ in rodent postmitotic tissues.

Rafael J. Yáñez-Muñoz, C. von Kalle, B. Korn, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Hematopoietic Repopulation, In Vivo, with Genetically Defined Clones Derived from HOXB4 Expressing ES-Cells.

S. Carotta, H. Beug, P. Steinlein, 2005 .

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

C. von Kalle, C. Eaves, H. Glimm, 2006, Blood.

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

A. Schambach, K. Kaufmann, M. Grez, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

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

H. Büning, Manfred Schmidt, M. Schmidt, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, M. Feuerer, Manfred Schmidt, 2015, The Journal of Immunology.

Cell Cycle Status of CD34+ Hemopoietic Stem Cells Determines Lentiviral Integration in Actively Transcribed and Development-related Genes.

C. von Kalle, I. Kasampalidis, N. Anagnou, 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB‐206 study

J. Tisdale, Manfred Schmidt, M. Hsieh, 2022, American journal of hematology.

Immunological and metabolic correction after lentiviral vector mediated haematopoietic stem cell gene therapy for ADA deficiency

K. Cornetta, D. Kohn, A. Thrasher, 2014 .

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

K. Cornetta, M. Hershfield, G. Crooks, 2021, The New England journal of medicine.

Lentiviral Vector Promoter is Decisive for Aberrant Transcript Formation.

C. von Kalle, R. Fronza, Manfred Schmidt, 2017, Human gene therapy.

Biffi Metachromatic Leukodystrophy Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits

Gianluigi Zanetti, Luca Biasco, Alessandro Aiuti, 2013 .

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

L. Naldini, C. Doglioni, T. Nichols, 2015, Science Translational Medicine.

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

David A. Williams, C. von Kalle, S. Karlsson, 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

Christof von Kalle, Richard Gabriel, Hanno Glimm, 2011, PloS one.

Update on gene therapy for chronic granulomatous disease

A. Jauch, B. Burwinkel, A. Thrasher, 2007 .

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

J. Prieto, B. Sangro, H. Petry, 2016, Journal of hepatology.

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

Raffaele Fronza, Saira Afzal, Manfred Schmidt, 2020, Molecular therapy. Methods & clinical development.

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

Christof von Kalle, Richard Gabriel, Hanno Glimm, 2012, Human gene therapy methods.

The LAM-PCR Method to Sequence LV Integration Sites.

Wei Wang, Richard Gabriel, Manfred Schmidt, 2016, Methods in molecular biology.

Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome.

C. von Kalle, L. Biasco, L. Naldini, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Comparison of Lentiviral and Sleeping Beauty Mediated αβ T Cell Receptor Gene Transfer

A. Field, C. Vink, A. Thrasher, 2013, PloS one.

Vector integration and tumorigenesis.

C. von Kalle, Manfred Schmidt, A. Deichmann, 2014, Human gene therapy.

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

C. von Kalle, A. Fischer, M. Cavazzana‐Calvo, 2005, Blood.

Pioneer Vector Integration and Tumorigenesis

Manfred Schmidt, C. Kalle, A. Deichmann, 2014 .

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

G. Zanetti, Enrico Rubagotti, A. Biffi, 2014 .

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

C. von Kalle, T. Kafri, P. Monahan, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

C. Adamsbaum, P. Bougnères, Manfred Schmidt, 2021, Human gene therapy.

Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver.

Manfred Schmidt, M. Penaud-Budloo, C. Kalle, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver.

Christof von Kalle, Manfred Schmidt, C. von Kalle, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, H. Glimm, Manfred Schmidt, 2012, Methods in molecular biology.

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

E. Henckaerts, R. Fronza, Manfred Schmidt, 2015, Journal of Virology.

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

H. Büning, Manfred Schmidt, Irene Gil-Farina, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, Manfred Schmidt, R. Gabriel, 2014, Journal of visualized experiments : JoVE.

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

Alaine T. Ruthe, Xuegong Zhang, C. von Kalle, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle (cid:1)

F. Rolling, Manfred Schmidt, M. Penaud-Budloo, 2008 .

345. Sensitive Molecular Detection of Various Stable Episomal Concatemeric and Integrated Forms of rAAV2 after Long-Term Expression In Vivo

H. Glimm, K. Zaoui, K. High, 2004 .

Fragile sites are preferential targets for integrations of MLV vectors in gene therapy

B. Kerem, M. Cavazzana‐Calvo, A. Bester, 2006, Gene Therapy.

METALLOTHIONEI NEXPRESSIO NI NOVARIA NCANCE RI NRELATIO NTO HISTOPATHOLOGICA LPARAMETER SAN DMOLECULA RMARKER SOF PROGNOSIS

F. Oesch, H. Pilch, J. Hengstler, 2001 .

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

J. Tolar, J. Bueren, H. Hanenberg, 2012, Human Gene Therapy.

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

C. von Kalle, A. Schambach, M. Dinauer, 2013, Human gene therapy. Clinical development.

Correction of Wiskott-Aldrich Syndrome by Hematopoietic Stem Cell Gene Therapy

J. Orange, W. Al-Herz, C. Klein, 2010 .

The Clonal Fate of Live Cells

R. Fronza, Wei Wang, Manfred Schmidt, 2017, Molecular Therapy: Methods & Clinical Development.

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

L. Kaderali, M. Bickel, Manfred Schmidt, 2014, Journal of Antimicrobial Chemotherapy.

Integration of retroviral vectors.

C. von Kalle, Manfred Schmidt, R. Gabriel, 2012, Current Opinion in Immunology.

Pacemaker cell characteristics of differentiated and HCN4-transduced human mesenchymal stem cells.

A. Draguhn, C. Bruehl, D. Hose, 2019, Life Science.

Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206

Weiliang Shi, J. Tisdale, Manfred Schmidt, 2019, Biology of Blood and Marrow Transplantation.

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

C. von Kalle, A. Ganser, D. Wicke, 2012, Human Gene Therapy Methods.

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

J. Tchinda, A. Jauch, M. Grez, 2015, Current gene therapy.

Modeling and Increasing the Genome Accessibility of Viral Integration Sites

Alessandro Aiuti, Steven J. Howe, Robin R. Ali, 2009 .

Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B

R. Dolmetsch, H. Thomsen, G. Foster, 2023, Blood advances.

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

Raffaele Fronza, Manfred Schmidt, Hanno Glimm, 2014, Nature Medicine.

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

E. Henckaerts, R. Fronza, Manfred Schmidt, 2014, BioTechniques.

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

David A. Williams, C. von Kalle, T. Neff, 2003, The Journal of clinical investigation.

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

Jürgen Weitz, Christof von Kalle, Benedikt Brors, 2017, EMBO molecular medicine.

Stable Long‐Term Blood Formation by Stem Cells in Murine Steady‐State Hematopoiesis

C. von Kalle, H. Glimm, C. Ball, 2012, Stem cells.

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

C. von Kalle, H. Glimm, C. Ball, 2008, Blood.

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

Beau R. Webber, C. von Kalle, D. Voytas, 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

J. Rasko, E. Vichinsky, G. Schiller, 2018, Blood.

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

Raffaele Fronza, Manfred Schmidt, Richard Gabriel, 2019, Briefings Bioinform..

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

S. Ylä-Herttuala, Manfred Schmidt, D. Schenkwein, 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, C. Brombin, L. Naldini, 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

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.

Y. Takeuchi, Heba Saadeh, A. Thrasher, 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

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

C. von Kalle, G. Zanetti, Enrico Rubagotti, 2011, Blood.

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

C. von Kalle, C. Di Serio, L. Naldini, 2009, The Journal of clinical investigation.