Maria Carmina Castiello


Area of interest:

Dr. Castiello graduated from Università degli Studi di Napoli Federico II with a Master of Science Degree in Medical Biotechnology in 2007 under the supervision of Prof. Cittadini, where she studied the role of anti-apoptotic and growth factors in cardiovascular diseases. She then moved to Milan reaching the Unit of Pathogenesis and Treatment of Immune and Bone Diseases at San Raffaele Telethon institute for Gene therapy (SR-Tiget) under the supervision of Dr. Villa. In 2011, she was a visiting fellow at the Department of Molecular Immunology at the Erasmus Medical Center in Rotterdam (The Netherlands). She earned her PhD from Vita-Salute San Raffaele University (Milan) in 2013 defining B lymphocyte and platelet defects in Wiskott-Aldrich Syndrome primary immunodeficiency and evaluating their correction in patients treated by lentiviral vector mediated gene therapy. She performed her postdoctoral studies at SR-Tiget on mechanisms underlying autoimmunity in immunodeficiency and innovative therapeutic approaches to correct immunodeficiencies. Her research was very productive receiving the “Excellence Research Award” from the American Society of Gene and Cell Therapy (2016, Washington DC, US). In 2019, she received a permanent position as Researcher at the CNR-Institute of Genetic and Biomedical Research (IRGB) in Milan. She is now coordinating projects on translational medicine including the development of genome editing approach to treat RAG1 deficiency and novel non-genotoxic conditioning regimens to improve the safety and outcome of hematopoietic stem cell transplants. She works with an excellent team of researchers and clinicians bringing to the clinic cutting-edge therapies for patients affected by rare genetic diseases.

Most significant publications:


Castiello, M C; Bosticardo, M; Sacchetti, N; Calzoni, E; Fontana, E; Yamazaki, Y; Draghici, E; Corsino, C; Bortolomai, I; Sereni, L; Yu, H H; Uva, P; Palchaudhuri, R; Scadden, D T; Villa, A; Notarangelo, L D

Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency Journal Article

In: J Allergy Clin Immunol, 147 (1), pp. 309–320, 2021.



Villa, A; Capo, V; Castiello, M C

Innovative Cell-Based Ŧherapies and Conditioning to Cure RAG Đeficiency Journal Article

In: Front Immunol, 11 , pp. 607926, 2020.



Sereni, L; Castiello, M C; Silvestre, D Di; Valle, P Della; 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; DÁngelo, A; Aiuti, A; Mauri, P; Villa, A

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

In: J Allergy Clin Immunol, 144 (3), pp. 825–838, 2019.



Capo, V; Castiello, M C; Fontana, E; Penna, S; Bosticardo, M; Draghici, E; Poliani, L P; Sergi, L Sergi; Rigoni, R; Cassani, B; Zanussi, M; Carrera, P; Uva, P; Dobbs, K; Sacchetti, N; Notarangelo, L D; van Til, N P; Wagemaker, G; Villa, A

Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation Journal Article

In: J Allergy Clin Immunol, 142 (3), pp. 928–941, 2018.


Sereni, L; Castiello, M C; Marangoni, F; Anselmo, A; di Silvestre, D; Motta, S; Draghici, E; Mantero, S; Thrasher, A J; Giliani, S; Aiuti, A; Mauri, P; Notarangelo, L D; Bosticardo, M; Villa, A

Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation Journal Article

In: J Allergy Clin Immunol, 142 (4), pp. 1272–1284, 2018.



Castiello, M C; Pala, F; Sereni, L; Draghici, E; Inverso, D; Sauer, A V; Schena, F; Fontana, E; Radaelli, E; Uva, P; Cervantes-Luevano, K E; Benvenuti, F; Poliani, P L; Iannacone, M; Traggiai, E; Villa, A; Bosticardo, M

In Vivo Chronic Stimulation Unveils Autoreactive Potential of Wiskott-Aldrich Syndrome Protein-Đeficient B Cells Journal Article

In: Front Immunol, 8 , pp. 490, 2017.



Castiello, Maria Carmina; Scaramuzza, Samantha; Pala, Francesca; Ferrua, Francesca; Uva, Paolo; Brigida, Immacolata; Sereni, Lucia; van der Burg, Mirjam; Ottaviano, Giorgio; Albert, Michael H; Roncarolo, Maria Grazia; Naldini, Luigi; Aiuti, Alessandro; Villa, Anna; Bosticardo, Marita

B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome Journal Article

In: The Journal of Allergy and Clinical Immunology, 136 (3), pp. 692––702.e2, 2015, ISSN: 1097-6825.

Abstract | Links | BibTeX


Castiello, Maria Carmina; Bosticardo, Marita; Pala, Francesca; Catucci, Marco; Chamberlain, Nicolas; van Zelm, Menno C; Driessen, Gertjan J; Pac, Malgorzata; Bernatowska, Ewa; Scaramuzza, Samantha; Aiuti, Alessandro; Sauer, Aisha V; Traggiai, Elisabetta; Meffre, Eric; Villa, Anna; van der Burg, Mirjam

Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans Journal Article

In: Journal of Autoimmunity, 50 , pp. 42–50, 2014, ISSN: 1095-9157.

Abstract | Links | BibTeX


Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome Journal Article

In: Science, 341 (6148), pp. 1233151, 2013.