• 12 Citations
  • 2 h-Index
20182019
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Research Output 2018 2019

  • 12 Citations
  • 2 h-Index
  • 5 Article
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Article
2019
1 Citation (Scopus)

A novel disorder involving dyshematopoiesis, inflammation, and HLH due to aberrant CDC42 function

Lam, M. T., Coppola, S., Krumbach, O. H. F., Prencipe, G., Insalaco, A., Cifaldi, C., Brigida, I., Zara, E., Scala, S., Di Cesare, S., Martinelli, S., Di Rocco, M., Pascarella, A., Niceta, M., Pantaleoni, F., Ciolfi, A., Netter, P., Carisey, A. F., Diehl, M., Akbarzadeh, M. & 30 others, Conti, F., Merli, P., Pastore, A., Levi Mortera, S., Camerini, S., Farina, L., Buchholzer, M., Pannone, L., Cao, T. N., Coban-Akdemir, Z. H., Jhangiani, S. N., Muzny, D. M., Gibbs, R. A., Basso-Ricci, L., Chiriaco, M., Dvorsky, R., Putignani, L., Carsetti, R., Janning, P., Stray-Pedersen, A., Erichsen, H. C., Rosti, V., Bracaglia, C., Palma, P., Finocchi, A., Locatelli, F., Cancrini, C., Aiuti, A., De Benedetti, F. & Tartaglia, M., Dec 2 2019, In : The Journal of experimental medicine. 216, 12, p. 2778-2799 22 p.

Research output: Contribution to journalArticle

Open Access
3 Citations (Scopus)
Open Access
Thalassemia
Mesenchymal Stromal Cells
Bone Marrow
Iron Overload
Hematopoietic Stem Cells
8 Citations (Scopus)

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
Wiskott-Aldrich Syndrome
Cell- and Tissue-Based Therapy
Genetic Therapy
Stem Cells
Wiskott-Aldrich Syndrome Protein
2018

Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

Scala, S., Basso-Ricci, L., Dionisio, F., Pellin, D., Giannelli, S., Salerio, FA., Leonardelli, L., Cicalese, MP., Ferrua, F., Aiuti, A. & Biasco, L., 2018, In : Nature Medicine. 24, 11, p. 1683-1690 8 p.

Research output: Contribution to journalArticle

Autologous Transplantation
Hematopoietic Stem Cells
Stem cells
Gene therapy
Transplants

T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

Brigida, I., Zoccolillo, M., Cicalese, MP., Pfajfer, L., Barzaghi, F., Scala, S., Oleaga-Quintas, C., Álvarez-Álvarez, JA., Sereni, L., Giannelli, S., Sartirana, C., Dionisio, F., Pavesi, L., Benavides-Nieto, M., Basso-Ricci, L., Capasso, P., Mazzi, B., Rosain, J., Marcus, N., Lee, YN. & 26 others, Somech, R., Degano, M., Raiola, G., Caorsi, R., Picco, P., Velez, MM., Khourieh, J., Arias, AA., Bousfiha, A., Issekutz, T., Issekutz, A., Boisson, B., Dobbs, K., Villa, A., Lombardo, A., Neven, B., Moshous, D., Casanova, JL., Franco, JL., Notarangelo, LD., Scielzo, C., Volpi, S., Dupré, L., Bustamante, J., Gattorno, M. & Aiuti, A., 2018, In : Blood. 132, 22, p. 2362-2374 13 p.

Research output: Contribution to journalArticle

T-cells
Germ-Line Mutation
T-Lymphocytes
Defects
T-Cell Antigen Receptor