Roncarolo, Maria Grazia

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

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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

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Reprogramming human T cell function and specificity with non-viral genome targeting

Roth, T. L., Puig-Saus, C., Yu, R., Shifrut, E., Carnevale, J., Li, P. J., Hiatt, J., Saco, J., Krystofinski, P., Li, H., et al. (2018) · Nature

CRISPR and Genetic EngineeringCAR-T cell therapy research

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Regulatory T cells: recommendations to simplify the nomenclature

Abbas, A. K., Benoist, C., Bluestone, J. A., Campbell, D. J., Ghosh, S., Hori, S., Jiang, S., Kuchroo, V. K., Mathis, D., Roncarolo, M. G., et al. (2013) · Nature Immunology

T-cell and B-cell ImmunologyPsoriasis: Treatment and Pathogenesis

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Gene Therapy

High, K. A., & Roncarolo, M. G. (2019) · New England Journal of Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

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Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Sessa, M., Lorioli, L., Fumagalli, F., Acquati, S., Redaelli, D., Baldoli, C., Canale, S., Lopez, I. D., Morena, F., Calabria, A., et al. (2016) · The Lancet

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

Aiuti, A., Roncarolo, M. G., & Naldini, L. (2017) · EMBO Molecular Medicine

Virus-based gene therapy researchCAR-T cell therapy research

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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

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In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells

Biasco, L., Scala, S., Basso Ricci, L., Dionisio, F., Baricordi, C., Calabria, A., Giannelli, S., Cieri, N., Barzaghi, F., Pajno, R., et al. (2015) · Science Translational Medicine

Virus-based gene therapy researchT-cell and B-cell Immunology

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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

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A novel function for FOXP3 in humans: intrinsic regulation of conventional T cells

McMurchy, A. N., Gillies, J., Gizzi, M. C., Riba, M., Garcia-Manteiga, J. M., Cittaro, D., Lazarevic, D., Di Nunzio, S., Piras, I. S., Bulfone, A., et al. (2013) · Blood

T-cell and B-cell ImmunologyPsoriasis: Treatment and Pathogenesis

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