Schambach, Axel

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

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., et al. (2014) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 412

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M. H., Schambach, A., Charrier, S., Galy, A., Thrasher, A. J., et al. (2009) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 387

Long noncoding RNA Chast promotes cardiac remodeling

Viereck, J., Kumarswamy, R., Foinquinos, A., Xiao, K., Avramopoulos, P., Kunz, M., Dittrich, M., Maetzig, T., Zimmer, K., Remke, J., et al. (2016) · Science Translational Medicine

Cardiac Fibrosis and RemodelingCancer-related molecular mechanisms research

Cited by 370

A Differentiation Checkpoint Limits Hematopoietic Stem Cell Self-Renewal in Response to DNA Damage

Wang, J., Sun, Q., Morita, Y., Jiang, H., Groß, A., Lechel, A., Hildner, K., Guachalla, L. M., Gompf, A., Hartmann, D., et al. (2012) · Cell

Hematopoietic Stem Cell TransplantationDNA Repair Mechanisms

Cited by 343

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., & Baum, C. (2008) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 271

Rapid and efficient generation of oligodendrocytes from human induced pluripotent stem cells using transcription factors

Ehrlich, M., Mozafari, S., Glatza, M., Starost, L., Velychko, S., Hallmann, A.-L., Cui, Q.-L., Schambach, A., Kim, K.-P., Bachelin, C., et al. (2017) · Proceedings of the National Academy of Sciences

Pluripotent Stem Cells ResearchNeurogenesis and neuroplasticity mechanisms

Cited by 257

Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy

Papapetrou, E. P., & Schambach, A. (2016) · Molecular Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 238

Large-scale hematopoietic differentiation of human induced pluripotent stem cells provides granulocytes or macrophages for cell replacement therapies

Lachmann, N., Ackermann, M., Frenzel, E., Liebhaber, S., Brennig, S., Happle, C., Hoffmann, D., Klimenkova, O., Lüttge, D., Buchegger, T., et al. (2015) · Stem Cell Reports

Pluripotent Stem Cells ResearchHematopoietic Stem Cell Transplantation

Cited by 238

Direct Reprogramming of Hepatic Myofibroblasts into Hepatocytes In Vivo Attenuates Liver Fibrosis

Song, G., Pacher, M., Balakrishnan, A., Yuan, Q., Tsay, H.-C., Yang, D., Reetz, J., Brandes, S., Dai, Z., Pützer, B. M., et al. (2016) · Cell Stem Cell

Liver physiology and pathologyPluripotent Stem Cells Research

Cited by 208

Inherited DOCK2 Deficiency in Patients with Early-Onset Invasive Infections

Dobbs, K., Domínguez Conde, C., Zhang, S.-Y., Parolini, S., Audry, M., Chou, J., Haapaniemi, E., Keles, S., Bilic, I., Okada, S., et al. (2015) · New England Journal of Medicine

Immunodeficiency and Autoimmune DisordersBlood disorders and treatments

Cited by 201

Biosafety Features of Lentiviral Vectors

Schambach, A., Zychlinski, D., Ehrnstroem, B., & Baum, C. (2013) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 173

Bioreactor-based mass production of human iPSC-derived macrophages enables immunotherapies against bacterial airway infections

Ackermann, M., Kempf, H., Hetzel, M., Hesse, C., Hashtchin, A. R., Brinkert, K., Schott, J. W., Haake, K., Kühnel, M. P., Glage, S., et al. (2018) · Nature Communications

Pluripotent Stem Cells ResearchMesenchymal stem cell research

Cited by 165

Bromo- and Extraterminal Domain Chromatin Regulators Serve as Cofactors for Murine Leukemia Virus Integration

Gupta, S. S., Maetzig, T., Maertens, G. N., Sharif, A., Rothe, M., Weidner-Glunde, M., Galla, M., Schambach, A., Cherepanov, P., & Schulz, T. F. (2013) · Journal of Virology

Virus-based gene therapy researchHIV Research and Treatment

Cited by 139

Improving Transcriptional Termination of Self-inactivating Gamma-retroviral and Lentiviral Vectors

Schambach, A., Galla, M., Maetzig, T., Loew, R., & Baum, C. (2007) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 127

Biosafety Challenges for Use of Lentiviral Vectors in Gene Therapy

Rothe, M., Modlich, U., & Schambach, A. (2014) · Current Gene Therapy

Virus-based gene therapy researchCRISPR and Genetic Engineering

Cited by 110

Viral and Synthetic RNA Vector Technologies and Applications

Schott, J. W., Morgan, M., Galla, M., & Schambach, A. (2016) · Molecular Therapy

RNA Interference and Gene DeliveryVirus-based gene therapy research

Cited by 76

A spatiotemporal atlas of mouse liver homeostasis and regeneration

Xu, J., Guo, P., Hao, S., Shangguan, S., Shi, Q., Volpe, G., Huang, K., Zuo, J., An, J., Yuan, Y., et al. (2024) · Nature Genetics

Liver physiology and pathologySingle-cell and spatial transcriptomics

Cited by 75

Preclinical evaluation of a novel lentiviral vector driving lineage-specific BCL11A knockdown for sickle cell gene therapy

Brendel, C., Negre, O., Rothe, M., Guda, S., Parsons, G., Harris, C., McGuinness, M., Abriss, D., Tsytsykova, A., Klatt, D., et al. (2020) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 72

Myeloid-Derived Growth Factor Protects Against Pressure Overload–Induced Heart Failure by Preserving Sarco/Endoplasmic Reticulum Ca 2+ -ATPase Expression in Cardiomyocytes

Korf-Klingebiel, M., Reboll, M. R., Polten, F., Weber, N., Jäckle, F., Wu, X., Kallikourdis, M., Kunderfranco, P., Condorelli, G., Giannitsis, E., et al. (2021) · Circulation

Cardiac Fibrosis and RemodelingCardiovascular Function and Risk Factors

Cited by 71

Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection

van der Loo, J. C. M., Swaney, W. P., Grassman, E., Terwilliger, A., Higashimoto, T., Schambach, A., Baum, C., Thrasher, A. J., Williams, D. A., Nordling, D. L., et al. (2012) · Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 34

CD24 targeting with NK‐CAR immunotherapy in testis, prostate, renal and (luminal‐type) bladder cancer and identification of direct CD24 interaction partners

Söhngen, C., Thomas, D. J., Skowron, M. A., Bremmer, F., Eckstein, M., Stefanski, A., Driessen, M. D., Wakileh, G. A., Stühler, K., Altevogt, P., et al. (2023) · The FEBS Journal

Immune Cell Function and InteractionCAR-T cell therapy research

Cited by 19

A Differentiation Checkpoint Limits Hematopoietic Stem Cell Self-Renewal in Response to DNA Damage

Wang, J., Sun, Q., Morita, Y., Jiang, H., Groß, A., Lechel, A., Hildner, K., Guachalla, L. M., Gompf, A., Hartmann, D., et al. (2014) · Cell

Hematopoietic Stem Cell TransplantationDNA Repair Mechanisms

Cited by 12