20152019

Research output per year

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Research Output

Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

Sereni, L., Castiello, M. C., Di Silvestre, D., Della Valle, P., Brombin, C., Ferrua, F., Cicalese, M. P., Pozzi, L., Migliavacca, M., Bernardo, M. E., Pignata, C., Farah, R., Notarangelo, L. D., Marcus, N., Cattaneo, L., Spinelli, M., Giannelli, S., Bosticardo, M., van Rossem, K., D'Angelo, A. & 3 others, Aiuti, A., Mauri, P. & Villa, A., Sep 2019, In : Journal of Allergy and Clinical Immunology. 144, 3, p. 825-838 14 p.

Research output: Contribution to journalArticle

Open Access
  • Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

    Ferrua, F., Cicalese, M. P., Galimberti, S., Giannelli, S., Dionisio, F., Barzaghi, F., Migliavacca, M., Bernardo, M. E., Calbi, V., Assanelli, A. A., Facchini, M., Fossati, C., Albertazzi, E., Scaramuzza, S., Brigida, I., Scala, S., Basso-Ricci, L., Pajno, R., Casiraghi, M., Canarutto, D. & 18 others, Salerio, F. A., Albert, M. H., Bartoli, A., Wolf, H. M., Fiori, R., Silvani, P., Gattillo, S., Villa, A., Biasco, L., Dott, C., Culme-Seymour, E. J., van Rossem, K., Atkinson, G., Valsecchi, M. G., Roncarolo, M. G., Ciceri, F., Naldini, L. & Aiuti, A., May 1 2019, In : The Lancet Haematology. 6, 5, p. e239-e253

    Research output: Contribution to journalArticle

    Open Access
  • Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study: The Lancet Haematology

    Ferrua, F., Cicalese, MP., Galimberti, S., Giannelli, S., Dionisio, F., Barzaghi, F., Migliavacca, M., Bernardo, ME., Calbi, V., Assanelli, AA., Facchini, M., Fossati, C., Albertazzi, E., Scaramuzza, S., Brigida, I., Scala, S., Basso-Ricci, L., Pajno, R., Casiraghi, M., Canarutto, D. & 18 others, Salerio, FA., Albert, MH., Bartoli, A., Wolf, HM., Fiori, R., Silvani, P., Gattillo, S., Villa, A., Biasco, L., Dott, C., Culme-Seymour, EJ., van Rossem, K., Atkinson, G., Valsecchi, MG., Roncarolo, MG., Ciceri, F., Naldini, L. & Aiuti, A., 2019, In : The Lancet Haematology. 6, 5, p. e239-e253

    Research output: Contribution to journalArticle

  • Stem Cell Modeling of Neuroferritinopathy Reveals Iron as a Determinant of Senescence and Ferroptosis during Neuronal Aging: Stem cell reports

    Cozzi, A., Orellana, DI., Santambrogio, P., Rubio, A., Cancellieri, C., Giannelli, S., Ripamonti, M., Taverna, S., Di Lullo, G., Rovida, E., Ferrari, M., Forni, GL., Fiorillo, C., Broccoli, V. & Levi, S., 2019, In : Stem Cell Reports. 13, 5, p. 832-846 15 p.

    Research output: Contribution to journalArticle

  • Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery

    Giannelli, SG., Luoni, M., Castoldi, V., Massimino, L., Cabassi, T., Angeloni, D., Demontis, GC., Leocani, L., Andreazzoli, M. & Broccoli, V., 2018, In : Human Molecular Genetics. 27, 5, p. 761-779 19 p.

    Research output: Contribution to journalArticle