20092019

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2019

A combined immunodeficiency with severe infections, inflammation, and allergy caused by ARPC1B deficiency: Journal of Allergy and Clinical Immunology

Volpi, S., Cicalese, MP., Tuijnenburg, P., Tool, ATJ., Cuadrado, E., Abu-Halaweh, M., Ahanchian, H., Alzyoud, R., Akdemir, ZC., Barzaghi, F., Blank, A., Boisson, B., Bottino, C., Brigida, I., Caorsi, R., Casanova, JL., Chiesa, S., Chinn, IK., Dückers, G., Enders, A. & 32 others, Erichsen, HC., Forbes, LR., Gambin, T., Gattorno, M., Karimiani, EG., Giliani, S., Gold, MS., Jacobsen, EM., Jansen, MH., King, JR., Laxer, RM., Lupski, JR., Mace, E., Marcenaro, S., Maroofian, R., Meijer, AB., Niehues, T., Notarangelo, LD., Orange, J., Pannicke, U., Pearson, C., Picco, P., Quinn, PJ., Schulz, A., Seeborg, F., Stray-Pedersen, A., Tawamie, H., van Leeuwen, EMM., Aiuti, A., Yeung, R., Schwarz, K. & Kuijpers, TW., 2019, In : Journal of Allergy and Clinical Immunology. 143, 6, p. 2296-2299 4 p.

Research output: Contribution to journalArticle

ALPS-like phenotype caused by ADA2 deficiency rescued by allogeneic hematopoietic stem cell transplantation

Barzaghi, F., Minniti, F., Mauro, M., De Bortoli, M., Balter, R., Bonetti, E., Zaccaron, A., Vitale, V., Omrani, M., Zoccolillo, M., Brigida, I., Cicalese, M. P., Degano, M., Hershfield, M. S., Aiuti, A., Bondarenko, A. V., Chinello, M. & Cesaro, S., Jan 1 2019, In : Frontiers in Immunology. 10, JAN

Research output: Contribution to journalArticle

Open Access

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

Tucci, F., Frittoli, M., Barzaghi, F., Calbi, V., Migliavacca, M., Ferrua, F., Fumagalli, F., Lorioli, L., Castagnaro, L., Facchini, M., Fossati, C., Zancan, S., Massariello, P., Manfredini, M., Consiglieri, G., Canarutto, D., Recupero, S., Calzatini, F., Casiraghi, M., Darin, S. & 14 others, Antonioli, G., Miniero, R., Fiori, R., Silvani, P., Zambelli, M., Marktel, S., Gattillo, S., Milani, R., Santoleri, L., Ciceri, F., Biffi, A., Cicalese, M. P., Bernardo, M. E. & Aiuti, A., Dec 1 2019, In : Bone Marrow Transplantation. 54, 12, p. 1995-2003 9 p.

Research output: Contribution to journalArticle

Open Access

Clinical, immunological, and molecular features of typical and atypical severe combined immunodeficiency: Report of the italian primary immunodeficiency network

Cirillo, E., Cancrini, C., Azzari, C., Martino, S., Martire, B., Pession, A., Tommasini, A., Naviglio, S., Finocchi, A., Consolini, R., Pierani, P., D’alba, I., Putti, M. C., Marzollo, A., Giardino, G., Prencipe, R., Esposito, F., Grasso, F., Scarselli, A., Di Matteo, G. & 15 others, Attardi, E., Ricci, S., Montin, D., Specchia, F., Barzaghi, F., Cicalese, M. P., Quaremba, G., Lougaris, V., Giliani, S., Locatelli, F., Rossi, P., Aiuti, A., Badolato, R., Plebani, A. & Pignata, C., 2019, In : Frontiers in Immunology. 10, AUG

Research output: Contribution to journalArticle

Clinical, immunological, and molecular features of typical and atypical severe combined immunodeficiency: Report of the Italian primary immunodeficiency network: Frontiers in Immunology

Cirillo, E., Cancrini, C., Azzari, C., Martino, S., Martire, B., Pession, A., Tommasini, A., Naviglio, S., Finocchi, A., Consolini, R., Pierani, P., D’alba, I., Putti, MC., Marzollo, A., Giardino, G., Prencipe, R., Esposito, F., Grasso, F., Scarselli, A., Di Matteo, G. & 15 others, Attardi, E., Ricci, S., Montin, D., Specchia, F., Barzaghi, F., Cicalese, MP., Quaremba, G., Lougaris, V., Giliani, S., Locatelli, F., Rossi, P., Aiuti, A., Badolato, R., Plebani, A. & Pignata, C., 2019, In : Frontiers in Immunology. 10

Research output: Contribution to journalArticle

Efficacy of the andresen activator before peak growth in class ii patients

Faccioni, P., de Santis, D., Luciano, U., Pancera, P., Sinigaglia, S., Iurlaro, A., Bertossi, D., Lucchese, A., Manuelli, M., Maestrini, S., Kumar, N., Zotti, F., Donadello, D., Ricciardi, G., Albanese, M. & Cicalese, M. P., Jan 1 2019, In : Journal of Biological Regulators and Homeostatic Agents. 33, 1, p. 1-7 7 p.

Research output: Contribution to journalArticle

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia: Nature Medicine

Marktel, S., Scaramuzza, S., Cicalese, MP., Giglio, F., Galimberti, S., Lidonnici, MR., Calbi, V., Assanelli, A., Bernardo, ME., Rossi, C., Calabria, A., Milani, R., Gattillo, S., Benedicenti, F., Spinozzi, G., Aprile, A., Bergami, A., Casiraghi, M., Consiglieri, G., Masera, N. & 18 others, D’Angelo, E., Mirra, N., Origa, R., Tartaglione, I., Perrotta, S., Winter, R., Coppola, M., Viarengo, G., Santoleri, L., Graziadei, G., Gabaldo, M., Valsecchi, MG., Montini, E., Naldini, L., Cappellini, MD., Ciceri, F., Aiuti, A. & Ferrari, G., 2019, In : Nat. Med.. 25, 2, p. 234-241 8 p.

Research output: Contribution to journalArticle

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

Marktel, S., Scaramuzza, S., Cicalese, M. P., Giglio, F., Galimberti, S., Lidonnici, M. R., Calbi, V., Assanelli, A., Bernardo, M. E., Rossi, C., Calabria, A., Milani, R., Gattillo, S., Benedicenti, F., Spinozzi, G., Aprile, A., Bergami, A., Casiraghi, M., Consiglieri, G., Masera, N. & 18 others, D’Angelo, E., Mirra, N., Origa, R., Tartaglione, I., Perrotta, S., Winter, R., Coppola, M., Viarengo, G., Santoleri, L., Graziadei, G., Gabaldo, M., Valsecchi, M. G., Montini, E., Naldini, L., Cappellini, M. D., Ciceri, F., Aiuti, A. & Ferrari, G., Jan 1 2019, (Accepted/In press) In : Nature Medicine.

Research output: Contribution to journalArticle

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

Marktel, S., Scaramuzza, S., Cicalese, M. P., Giglio, F., Galimberti, S., Lidonnici, M. R., Calbi, V., Assanelli, A., Bernardo, M. E., Rossi, C., Calabria, A., Milani, R., Gattillo, S., Benedicenti, F., Spinozzi, G., Aprile, A., Bergami, A., Casiraghi, M., Consiglieri, G., Masera, N. & 18 others, D’Angelo, E., Mirra, N., Origa, R., Tartaglione, I., Perrotta, S., Winter, R., Coppola, M., Viarengo, G., Santoleri, L., Graziadei, G., Gabaldo, M., Valsecchi, M. G., Montini, E., Naldini, L., Cappellini, M. D., Ciceri, F., Aiuti, A. & Ferrari, G., 2019, In : Nature Medicine. 25, 2, p. 234-241 8 p.

Research output: Contribution to journalArticle

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

Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies: Frontiers in immunology

Cifaldi, C., Brigida, I., Barzaghi, F., Zoccolillo, M., Ferradini, V., Petricone, D., Cicalese, MP., Lazarevic, D., Cittaro, D., Omrani, M., Attardi, E., Conti, F., Scarselli, A., Chiriaco, M., Di Cesare, S., Licciardi, F., Davide, M., Ferrua, F., Canessa, C., Pignata, C. & 20 others, Giliani, S., Ferrari, S., Fousteri, G., Barera, G., Merli, P., Palma, P., Cesaro, S., Gattorno, M., Trizzino, A., Moschese, V., Chini, L., Villa, A., Azzari, C., Finocchi, A., Locatelli, F., Rossi, P., Sangiuolo, F., Aiuti, A., Cancrini, C. & Di Matteo, G., 2019, In : Frontiers in Immunology. 10

Research output: Contribution to journalArticle

2018

Circulating follicular helper and follicular regulatory T cells are severely compromised in human CD40 deficiency: A case report

Cicalese, M. P., Gerosa, J., Baronio, M., Montin, D., Licciardi, F., Soresina, A., Dellepiane, R. M., Miano, M., Baselli, L. A., Volpi, S., Dufour, C., Plebani, A., Aiuti, A., Lougaris, V. & Fousteri, G., Aug 6 2018, In : Frontiers in Immunology. 9, AUG, 1761.

Research output: Contribution to journalArticle

Circulating follicular helper and follicular regulatory T cells are severely compromised in human CD40 deficiency: A case report

Cicalese, MP., Gerosa, J., Baronio, M., Montin, D., Licciardi, F., Soresina, A., Dellepiane, RM., Miano, M., Baselli, LA., Volpi, S., Dufour, C., Plebani, A., Aiuti, A., Lougaris, V. & Fousteri, G., 2018, In : Frontiers in Immunology. 9, 1761.

Research output: Contribution to journalArticle

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

First occurrence of plasmablastic lymphoma in adenosine deaminase-deficient severe combined immunodeficiency disease patient and review of the literature

Migliavacca, M., Assanelli, A., Ponzoni, M., Pajno, R., Barzaghi, F., Giglio, F., Ferrua, F., Frittoli, M., Brigida, I., Dionisio, F., Nicoletti, R., Casiraghi, M., Roncarolo, MG., Doglioni, C., Peccatori, J., Ciceri, F., Cicalese, MP. & Aiuti, A., 2018, In : Frontiers in Immunology. 9, 113.

Research output: Contribution to journalArticle

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Cicalese, MP., Ferrua, F., Castagnaro, L., Rolfe, K., De Boever, E., Reinhardt, RR., Appleby, J., Roncarolo, MG. & Aiuti, A., 2018, In : Molecular Therapy. 26, 3, p. 917-931 15 p.

Research output: Contribution to journalArticle

Gene therapy in rare diseases: The benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID

Stirnadel-Farrant, H., Kudari, M., Garman, N., Imrie, J., Chopra, B., Giannelli, S., Gabaldo, M., Corti, A., Zancan, S., Aiuti, A., Cicalese, MP., Batta, R., Appleby, J., Davinelli, M. & Ng, P., 2018, In : Orphanet Journal of Rare Diseases. 13, 1, 49.

Research output: Contribution to journalArticle

Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome

Cervantes-Luevano, K. E., Caronni, N., Castiello, M. C., Fontana, E., Piperno, G. M., Naseem, A., Uva, P., Bosticardo, M., Marcovecchio, G. E., Notarangelo, L. D., Cicalese, M. P., Aiuti, A., Villa, A. & Benvenuti, F., Nov 1 2018, In : Journal of Allergy and Clinical Immunology. 142, 5, p. 1605-1617.e4

Research output: Contribution to journalArticle

Neutrophils drive type I interferon production and autoantibodies in patients with Wiskott-Aldrich syndrome

Cervantes-Luevano, KE., Caronni, N., Castiello, MC., Fontana, E., Piperno, GM., Naseem, A., Uva, P., Bosticardo, M., Marcovecchio, GE., Notarangelo, LD., Cicalese, MP., Aiuti, A., Villa, A. & Benvenuti, F., 2018, In : Journal of Allergy and Clinical Immunology. 142, 5, p. 1605–1617.e4

Research output: Contribution to journalArticle

Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis

Tucci, F., Calbi, V., Barzaghi, F., Migliavacca, M., Ferrua, F., Bernardo, ME., Canarutto, D., Consiglieri, G., Recupero, S., Calzatini, F., Gabaldo, M., Lucano, C., Casiraghi, M., Darin, S., Dionisio, F., Marktel, S., Cestone, E., Finazzi, R., Mieli-Vergani, G., Boeri, E. & 5 others, Appleby, J., Abd Elaziz, D., Ciceri, F., Aiuti, A. & Cicalese, MP., 2018, In : Hepatology. 68, 6, p. 2434-2437 4 p.

Research output: Contribution to journalArticle

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

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

Calbi, V., Fumagalli, F., Consiglieri, G., Penati, R., Acquati, S., Redaelli, D., Attanasio, V., Marcella, F., Cicalese, MP., Migliavacca, M., Barzaghi, F., Ferrua, F., Assanelli, A., Silvani, P., Zoccolillo, M., Chidini, G., Chiesa, R., Arora, R., Ciotti, F., Sarzana, M. & 10 others, Antonioli, G., Baldoli, C., Morena, F., Martino, S., Ardissino, GL., Natali Sora, MG., Naldini, L., Ciceri, F., Aiuti, A. & Bernardo, ME., 2018, In : Bone Marrow Transplantation. 53, 3, p. 913-917 5 p.

Research output: Contribution to journalArticle

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

Calbi, V., Fumagalli, F., Consiglieri, G., Penati, R., Acquati, S., Redaelli, D., Attanasio, V., Marcella, F., Cicalese, M. P., Migliavacca, M., Barzaghi, F., Ferrua, F., Assanelli, A., Silvani, P., Zoccolillo, M., Chidini, G., Chiesa, R., Arora, R., Ciotti, F., Sarzana, M. & 10 others, Antonioli, G., Baldoli, C., Morena, F., Martino, S., Ardissino, G. L., Sora, M. G. N., Naldini, L., Ciceri, F., Aiuti, A. & Bernardo, M. E., 2018, In : Bone Marrow Transplantation. 53, 7, p. 913-917 5 p.

Research output: Contribution to journalArticle

2017

Beta2 integrins are required for follicular helper T cell differentiation in humans

Gerosa, J., Lougaris, V., Baronio, M., Plebani, A., Cicalese, MP. & Fousteri, G., 2017, In : Clinical Immunology. 180, 4, p. 60-62 3 p.

Research output: Contribution to journalArticle

Deconstructing IgG4-related disease involvement of midline structures: Comparison to common mimickers

Lanzillotta, M., Campochiaro, C., Trimarchi, M., Arrigoni, G., Gerevini, S., Milani, R., Bozzolo, E., Biafora, M., Venturini, E., Cicalese, MP., Stone, JH., Sabbadini, MG. & Della-Torre, E., 2017, In : Modern Rheumatology. 27, 4, p. 638-645 8 p.

Research output: Contribution to journalArticle

2016

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

Brigida, I., Scaramuzza, S., Lazarevic, D., Cittaro, D., Ferrua, F., Leonardelli, L., Alessio, M., Forma, O., Lanzani, C., Viarengo, G., Ciceri, F., Jankovic, M., Pesce, F., Aiuti, A. & Cicalese, M., 2016, (Accepted/In press) In : Journal of Allergy and Clinical Immunology.

Research output: Contribution to journalArticle

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

Brigida, I., Scaramuzza, S., Lazarevic, D., Cittaro, D., Ferrua, F., Leonardelli, L., Alessio, M., Forma, O., Lanzani, C., Viarengo, G., Ciceri, F., Jankovic, M., Pesce, F., Aiuti, A. & Cicalese, M. P., 2016, (Accepted/In press) In : Journal of Allergy and Clinical Immunology.

Research output: Contribution to journalArticle

A novel genomic inversion in Wiskott-Aldrich–associated autoinflammation

Brigida, I., Scaramuzza, S., Lazarevic, D., Cittaro, D., Ferrua, F., Leonardelli, L., Alessio, M., Forma, O., Lanzani, C., Viarengo, G., Ciceri, F., Jankovic, M., Pesce, F., Aiuti, A. & Cicalese, M. P., Aug 1 2016, In : Journal of Allergy and Clinical Immunology. 138, 2, p. 619 - 622.e7

Research output: Contribution to journalArticle

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, M. C., Canale, S., Lopez, I. D., Morena, F., Calabria, A., Fiori, R., Silvani, P., Rancoita, P. M. V., Gabaldo, M., Benedicenti, F., Antonioli, G., Assanelli, A., Cicalese, M. P., del Carro, U., Sora, M. G. N. & 9 others, Martino, S., Quattrini, A., Montini, E., Di Serio, C., Ciceri, F., Roncarolo, M. G., Aiuti, A., Naldini, L. & Biffi, A., 2016, In : The Lancet. 388, 10043, p. 476-487 12 p.

Research output: Contribution to journalArticle

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease

Migliavacca, M., Assanelli, A., Ferrua, F., Cicalese, M., Biffi, A., Frittoli, M. C., Silvani, P., Chidini, G., Calderini, E. L., Mandelli, A., Camporesi, A., Milani, R., Farinelli, G., Nicoletti, R., Ciceri, F., Aiuti, A. & Bernardo, M. E., 2016, In : Journal of Allergy and Clinical Immunology. 137, 6, p. 1913-1915e2 3 p.

Research output: Contribution to journalArticle

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease

Migliavacca, M., Assanelli, A., Ferrua, F., Cicalese, M. P., Biffi, A., Frittoli, M., Silvani, P., Chidini, G., Calderini, E., Mandelli, A., Camporesi, A., Milani, R., Farinelli, G., Nicoletti, R., Ciceri, F., Aiuti, A. & Bernardo, M. E., 2016, (Accepted/In press) In : Journal of Allergy and Clinical Immunology.

Research output: Contribution to journalArticle

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

Cicalese, M. P., Ferrua, F., Castagnaro, L., Pajno, R., Barzaghi, F., Giannelli, S., Dionisio, F., Brigida, I., Bonopane, M., Casiraghi, M., Tabucchi, A., Carlucci, F., Grunebaum, E., Adeli, M., Bredius, R. G., Puck, J. M., Stepensky, P., Tezcan, I., Rolfe, K., De Boever, E. & 5 others, Reinhardt, R. R., Appleby, J., Ciceri, F., Roncarolo, M. G. & Aiuti, A., Jul 7 2016, In : Blood. 128, 1, p. 45-54 10 p.

Research output: Contribution to journalArticle

2015

Abnormalities of acid-base balance and predisposition to metabolic acidosis in Metachromatic Leukodystrophy patients

Lorioli, L., Cicalese, M. P., Silvani, P., Assanelli, A., Salvo, I., Mandelli, A., Fumagalli, F., Fiori, R., Ciceri, F., Aiuti, A., Sessa, M., Roncarolo, M. G., Lanzani, C. & Biffi, A., 2015, In : Molecular Genetics and Metabolism. 115, 1, p. 48-52 5 p.

Research output: Contribution to journalArticle

Central venous access devices in pediatric malignancies: A position paper of Italian association of pediatric hematology and oncology

Crocoli, A., Tornesello, A., Pittiruti, M., Barone, A., Muggeo, P., Inserra, A., Molinari, A. C., Grillenzoni, V., Durante, V., Cicalese, M. P., Zanazzo, G. A. & Cesaro, S., Mar 1 2015, In : Journal of Vascular Access. 16, 2, p. 130-136 7 p.

Research output: Contribution to journalArticle

Clinical applications of gene therapy for primary immunodeficiencies

Cicalese, M. P. & Aiuti, A., Apr 1 2015, In : Human Gene Therapy. 26, 4, p. 210-219 10 p.

Research output: Contribution to journalArticle

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

Cossu, G., Previtali, S. C., Napolitano, S., Cicalese, M. P., Tedesco, F. S., Nicastro, F., Noviello, M., Roostalu, U., Natali Sora, M. G., Scarlato, M., De Pellegrin, M., Godi, C., Giuliani, S., Ciotti, F., Tonlorenzi, R., Lorenzetti, I., Rivellini, C., Benedetti, S., Gatti, R., Marktel, S. & 13 others, Mazzi, B., Tettamanti, A., Ragazzi, M., Imro, M. A., Marano, G., Ambrosi, A., Fiori, R., Sormani, M. P., Bonini, C., Venturini, M., Politi, L. S., Torrente, Y. & Ciceri, F., Dec 1 2015, In : EMBO Molecular Medicine. 7, 12, p. 1513-1528 16 p.

Research output: Contribution to journalArticle

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., Giannelli, S., Neduva, V., Dow, D. J., Schmidt, M., Von Kalle, C., Roncarolo, M. G., Ciceri, F., Vicard, P., Wit, E., Di Serio, C. & 2 others, Naldini, L. & Aiuti, A., Dec 3 2015, In : Cell Stem Cell. 19, 1, p. 107 - 119 13 p.

Research output: Contribution to journalArticle

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., Giannelli, S., Neduva, V., Dow, D. J., Schmidt, M., Von Kalle, C., Roncarolo, M. G., Ciceri, F., Vicard, P., Wit, E., Di Serio, C. & 2 others, Naldini, L. & Aiuti, A., Dec 3 2015, (Accepted/In press) In : Cell Stem Cell.

Research output: Contribution to journalArticle

Successful use of long-Acting octreotide for intractable chronic gastrointestinal bleeding in children

O'Meara, M., Cicalese, M. P., Bordugo, A., Ambrosi, A., Hadzic, N. & Mieli-Vergani, G., Jan 13 2015, In : Journal of Pediatric Gastroenterology and Nutrition. 60, 1, p. 48-53 6 p.

Research output: Contribution to journalArticle

Treatment of chronic hepatitis C in children with pegylated interferon and ribavirin: the impact of dose

Tufano, M., Cicalese, M. P., Spagnuolo, M. I., Terlizzi, V. & Iorio, R., Jan 1 2015, In : Acta Gastro-Enterologica Belgica. 78, p. 8-11 4 p.

Research output: Contribution to journalArticle

2014

B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients

Brigida, I., Sauer, A. V., Ferrua, F., Giannelli, S., Scaramuzza, S., Pistoia, V., Castiello, M. C., Barendregt, B. H., Cicalese, M. P., Casiraghi, M., Brombin, C., Puck, J., Müller, K., Notarangelo, L. D., Montin, D., Van Montfrans, J. M., Roncarolo, M. G., Traggiai, E., Van Dongen, J. J. M., Van Der Burg, M. & 1 others, Aiuti, A., Mar 2014, In : Journal of Allergy and Clinical Immunology. 133, 3

Research output: Contribution to journalArticle

Inflammation converts human mesoangioblasts into targets of alloreactive immune responses: Implications for allogeneic cell therapy of DMD

Noviello, M., Tedesco, F. S., Bondanza, A., Tonlorenzi, R., Rosaria Carbone, M., Gerli, M. F. M., Marktel, S., Napolitano, S., Cicalese, M. P., Ciceri, F., Peretti, G., Cossu, G. & Bonini, C., 2014, In : Molecular Therapy. 22, 7, p. 1342-1352 11 p.

Research output: Contribution to journalArticle

Intergenerational and intrafamilial phenotypic variability in 22q11.2 Deletion syndrome subjects

Cirillo, E., Giardino, G., Gallo, V., Puliafito, P., Azzari, C., Bacchetta, R., Cardinale, F., Cicalese, M. P., Consolini, R., Martino, S., Martire, B., Molinatto, C., Plebani, A., Scarano, G., Soresina, A., Cancrini, C., Rossi, P., Digilio, M. C. & Pignata, C., Jan 2 2014, In : BMC Medical Genetics. 15, 1, 1.

Research output: Contribution to journalArticle

Risk of seizures in children receiving busulphan-containing regimens for stem cell transplantation

Caselli, D., Rosati, A., Faraci, M., Podda, M., Ripaldi, M., Longoni, D., Cesaro, S., Lo Nigro, L., Paolicchi, O., Maximova, N., Menconi, M. C., Ziino, O., Cicalese, M. P., Santarone, S., Nesi, F., Aricò, M., Locatelli, F. & Prete, A., Feb 2014, In : Biology of Blood and Marrow Transplantation. 20, 2, p. 282-285 4 p.

Research output: Contribution to journalArticle

Single-day trimethoprim/sulfamethoxazole prophylaxis for pneumocystis pneumonia in children with cancer

Caselli, D., Petris, M. G., Rondelli, R., Carraro, F., Colombini, A., Muggeo, P., Ziino, O., Melchionda, F., Russo, G., Pierani, P., Soncini, E., Desantis, R., Zanazzo, G., Barone, A., Cesaro, S., Cellini, M., Mura, R., Milano, G. M., Meazza, C., Cicalese, M. P. & 4 others, Tropia, S., De Masi, S., Castagnola, E. & Aricò, M., Feb 2014, In : Journal of Pediatrics. 164, 2

Research output: Contribution to journalArticle

2013

Guidelines for the use of long-term central venous catheter in children with hemato-oncological disorders. on behalf of supportive therapy working group of Italian Association of Pediatric Hematology and Oncology (AIEOP)

Carraro, F., Cicalese, M. P., Cesaro, S., De Santis, R., Zanazzo, G., Tornesello, A., Giordano, P., Bergadano, A. & Giacchino, M., Oct 2013, In : Annals of Hematology. 92, 10, p. 1405-1412 8 p.

Research output: Contribution to journalArticle

Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity

Goudy, K., Aydin, D., Barzaghi, F., Gambineri, E., Vignoli, M., Mannurita, S. C., Doglioni, C., Ponzoni, M., Cicalese, M. P., Assanelli, A., Tommasini, A., Brigida, I., Dellepiane, R. M., Martino, S., Olek, S., Aiuti, A., Ciceri, F., Roncarolo, M. G. & Bacchetta, R., Mar 2013, In : Clinical Immunology. 146, 3, p. 248-261 14 p.

Research output: Contribution to journalArticle

La mucosite post-chemioterapia in bambini sottoposti a terapia genica, l'esperienza del San Raffaele di Milano

Buzzi, F., Gironi, R., Moser, C., Agazzi, S., Nunez Paiva, L., Guillermo Falcon, L., Matozzo, V., Soliman, C., Frittoli, M., Lorioli, L., Cicalese, M. P., Ciceri, F., Aiuti, A. & Biffi, A., Jun 2013, In : Pediatric Reports. 5, SUPPL.1, p. 109-110 2 p.

Research output: Contribution to journalArticle