Bartholomae, Cynthia C.

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

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

Enhancer hijacking activates GFI1 family oncogenes in medulloblastoma

Northcott, P. A., Lee, C., Zichner, T., Stütz, A. M., Erkek, S., Kawauchi, D., Shih, D. J. H., Hovestadt, V., Zapatka, M., Sturm, D., et al. (2014) · Nature

Glioma Diagnosis and TreatmentChromatin Remodeling and Cancer

Cited by 642

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

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

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

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

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

Lentiviral Hematopoietic Cell Gene Therapy for X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Bougnères, P., Schmidt, M., Von Kalle, C., Fischer, A., Cavazzana-Calvo, M., & Aubourg, P. (2012) · Methods in Enzymology

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 123