Schmidt, Manfred

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

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science

Virus-based gene therapy researchCAR-T cell therapy research

Cited by 3,587

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

Hacein-Bey-Abina, S., von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.-L., Fraser, C. C., Cavazzana-Calvo, M., et al. (2003) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 1,867

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

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 1,508

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

Howe, S. J., Mansour, M. R., Schwarzwaelder, K., Bartholomae, C., Hubank, M., Kempski, H., Brugman, M. H., Pike-Overzet, K., Chatters, S. J., de Ridder, D., et al. (2008) · Journal of Clinical Investigation

Virus-based gene therapy researchCAR-T cell therapy research

Cited by 1,286

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 1,180

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

Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science

Virus-based gene therapy researchCAR-T cell therapy research

Cited by 1,025

Dissecting the genomic complexity underlying medulloblastoma

Jones, D. T. W., Jäger, N., Kool, M., Zichner, T., Hutter, B., Sultan, M., Cho, Y.-J., Pugh, T. J., Hovestadt, V., Stütz, A. M., et al. (2012) · Nature

Glioma Diagnosis and TreatmentCancer Genomics and Diagnostics

Cited by 839

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

Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M., et al. (2009) · Journal of Clinical Investigation

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 571

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

Braun, C. J., Boztug, K., Paruzynski, A., Witzel, M., Schwarzer, A., Rothe, M., Modlich, U., Beier, R., Göhring, G., Steinemann, D., et al. (2014) · Science Translational Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 562

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

Boztug, K., Schmidt, M., Schwarzer, A., Banerjee, P. P., Díez, I. A., Dewey, R. A., Böhm, M., Nowrouzi, A., Ball, C. R., Glimm, H., et al. (2010) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 532

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

Gabriel, R., Lombardo, A., Arens, A., Miller, J. C., Genovese, P., Kaeppel, C., Nowrouzi, A., Bartholomae, C. C., Wang, J., Friedman, G., et al. (2011) · Nature Biotechnology

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 526

Effective gene therapy with nonintegrating lentiviral vectors

Yáñez-Muñoz, R. J., Balaggan, K. S., MacNeil, A., Howe, S. J., Schmidt, M., Smith, A. J., Buch, P., MacLaren, R. E., Anderson, P. N., Barker, S. E., et al. (2006) · Nature Medicine

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 460

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

Schmidt, M., Schwarzwaelder, K., Bartholomae, C., Zaoui, K., Ball, C., Pilz, I., Braun, S., Glimm, H., & von Kalle, C. (2007) · Nature Methods

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 328

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

Dieter, S. M., Ball, C. R., Hoffmann, C. M., Nowrouzi, A., Herbst, F., Zavidij, O., Abel, U., Arens, A., Weichert, W., Brand, K., et al. (2011) · Cell stem cell

Cancer Cells and MetastasisDevelopmental Biology and Gene Regulation

Cited by 297

TALEN-based Gene Correction for Epidermolysis Bullosa

Osborn, M. J., Starker, C. G., McElroy, A. N., Webber, B. R., Riddle, M. J., Xia, L., DeFeo, A. P., Gabriel, R., Schmidt, M., Von Kalle, C., et al. (2013) · Molecular Therapy

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 258

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

Biasco, L., Pellin, D., Scala, S., Dionisio, F., Basso-Ricci, L., Leonardelli, L., Scaramuzza, S., Baricordi, C., Ferrua, F., Cicalese, M. P., et al. (2016) · Cell stem cell

Hematopoietic Stem Cell TransplantationVirus-based gene therapy research

Cited by 243

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

Themis, M., Waddington, S. N., Schmidt, M., von Kalle, C., Wang, Y., Al-Allaf, F., Gregory, L. G., Nivsarkar, M., Themis, M., Holder, M. V., et al. (2005) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 243

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

Osborn, M. J., Webber, B. R., Knipping, F., Lonetree, C.-L., Tennis, N., DeFeo, A. P., McElroy, A. N., Starker, C. G., Lee, C., Merkel, S., et al. (2016) · Molecular Therapy

CRISPR and Genetic EngineeringCAR-T cell therapy research

Cited by 201

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

Cesana, D., Ranzani, M., Volpin, M., Bartholomae, C., Duros, C., Artus, A., Merella, S., Benedicenti, F., Sergi Sergi, L., Sanvito, F., et al. (2014) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 189

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

Gil-Farina, I., Fronza, R., Kaeppel, C., Lopez-Franco, E., Ferreira, V., D'Avola, D., Benito, A., Prieto, J., Petry, H., Gonzalez-Aseguinolaza, G., et al. (2016) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 179

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

Cantore, A., Ranzani, M., Bartholomae, C. C., Volpin, M., Valle, P. D., Sanvito, F., Sergi, L. S., Gallina, P., Benedicenti, F., Bellinger, D., et al. (2015) · Science Translational Medicine

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 158

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

Hsieh, M. M., Bonner, M., Pierciey, F. J., Uchida, N., Rottman, J., Demopoulos, L., Schmidt, M., Kanter, J., Walters, M. C., Thompson, A. A., et al. (2020) · Blood Advances

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 152

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

Mátrai, J., Cantore, A., Bartholomae, C. C., Annoni, A., Wang, W., Acosta-Sanchez, A., Samara-Kuko, E., De Waele, L., Ma, L., Genovese, P., et al. (2011) · Hepatology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 139

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

Negre, O., Bartholomae, C., Beuzard, Y., Cavazzana, M., Christiansen, L., Courne, C., Deichmann, A., Denaro, M., Dreuzy, E., Finer, M., et al. (2014) · Current Gene Therapy

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 126