Maria Carmina Castiello

@article{pmid32387109,

title = {Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency},

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

year  = {2021},

date = {2021-01-01},

journal = {J Allergy Clin Immunol},

volume = {147},

number = {1},

pages = {309--320},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid33329604,

title = {Innovative Cell-Based Ŧherapies and Conditioning to Cure RAG Đeficiency},

author = {A Villa and V Capo and M C Castiello},

year  = {2020},

date = {2020-01-01},

journal = {Front Immunol},

volume = {11},

pages = {607926},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid30926529,

title = {Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome},

author = {L Sereni and M C Castiello and D Di Silvestre and P Della Valle and C Brombin and F Ferrua and M P Cicalese and L Pozzi and M Migliavacca and M E Bernardo and C Pignata and R Farah and L D Notarangelo and N Marcus and L Cattaneo and M Spinelli and S Giannelli and M Bosticardo and K van Rossem and A DÁngelo and A Aiuti and P Mauri and A Villa},

year  = {2019},

date = {2019-01-01},

journal = {J Allergy Clin Immunol},

volume = {144},

number = {3},

pages = {825--838},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid29241731,

title = {Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation},

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

year  = {2018},

date = {2018-01-01},

journal = {J Allergy Clin Immunol},

volume = {142},

number = {3},

pages = {928--941},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid29421274,

title = {Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation},

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

year  = {2018},

date = {2018-01-01},

journal = {J Allergy Clin Immunol},

volume = {142},

number = {4},

pages = {1272--1284},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid28512459,

title = {In Vivo Chronic Stimulation Unveils Autoreactive Potential of Wiskott-Aldrich Syndrome Protein-Đeficient B Cells},

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

year  = {2017},

date = {2017-01-01},

journal = {Front Immunol},

volume = {8},

pages = {490},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{castiello_b-cell_2015,

title = {B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome},

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

doi = {10.1016/j.jaci.2015.01.035},

issn = {1097-6825},

year  = {2015},

date = {2015-09-01},

journal = {The Journal of Allergy and Clinical Immunology},

volume = {136},

number = {3},

pages = {692----702.e2},

abstract = {BACKGROUND: Wiskott-Aldrich syndrome (WAS) is a severe X-linked immunodeficiency characterized by microthrombocytopenia, eczema, recurrent infections, and susceptibility to autoimmunity and lymphomas. Hematopoietic stem cell transplantation is the treatment of choice; however, administration of WAS gene-corrected autologous hematopoietic stem cells has been demonstrated as a feasible alternative therapeutic approach. 

OBJECTIVE: Because B-cell homeostasis is perturbed in patients with WAS and restoration of immune competence is one of the main therapeutic goals, we have evaluated reconstitution of the B-cell compartment in 4 patients who received autologous hematopoietic stem cells transduced with lentiviral vector after a reduced-intensity conditioning regimen combined with anti-CD20 administration. 

METHODS: We evaluated B-cell counts, B-cell subset distribution, B cell-activating factor and immunoglobulin levels, and autoantibody production before and after gene therapy (GT). WAS gene transfer in B cells was assessed by measuring vector copy numbers and expression of Wiskott-Aldrich syndrome protein. 

RESULTS: After lentiviral vector-mediated GT, the number of transduced B cells progressively increased in the peripheral blood of all patients. Lentiviral vector-transduced progenitor cells were able to repopulate the B-cell compartment with a normal distribution of B-cell subsets both in bone marrow and the periphery, showing a WAS protein expression profile similar to that of healthy donors. In addition, after GT, we observed a normalized frequency of autoimmune-associated CD19(+)CD21(-)CD35(-) and CD21(low) B cells and a reduction in B cell-activating factor levels. Immunoglobulin serum levels and autoantibody production improved in all treated patients. 

CONCLUSIONS: We provide evidence that lentiviral vector-mediated GT induces transgene expression in the B-cell compartment, resulting in ameliorated B-cell development and functionality and contributing to immunologic improvement in patients with WAS.},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{castiello_wiskott-aldrich_2014,

title = {Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans},

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

doi = {10.1016/j.jaut.2013.10.006},

issn = {1095-9157},

year  = {2014},

date = {2014-05-01},

journal = {Journal of Autoimmunity},

volume = {50},

pages = {42--50},

abstract = {Wiskott-Aldrich Syndrome protein (WASp) regulates the cytoskeleton in hematopoietic cells and mutations in its gene cause the Wiskott-Aldrich Syndrome (WAS), a primary immunodeficiency with microthrombocytopenia, eczema and a higher susceptibility to develop tumors. Autoimmune manifestations, frequently observed in WAS patients, are associated with an increased risk of mortality and still represent an unsolved aspect of the disease. B cells play a crucial role both in immune competence and self-tolerance and defects in their development and function result in immunodeficiency and/or autoimmunity. We performed a phenotypical and molecular analysis of central and peripheral B-cell compartments in WAS pediatric patients. We found a decreased proportion of immature B cells in the bone marrow correlating with an increased presence of transitional B cells in the periphery. These results could be explained by the defective migratory response of WAS B cells to SDF-1α, essential for the retention of immature B cells in the BM. In the periphery, we observed an unusual expansion of CD21(low) B-cell population and increased plasma BAFF levels that may contribute to the high susceptibility to develop autoimmune manifestations in WAS patients. WAS memory B cells were characterized by a reduced in vivo proliferation, decreased somatic hypermutation and preferential usage of IGHV4-34, an immunoglobulin gene commonly found in autoreactive B cells. In conclusion, our findings demonstrate that WASp-deficiency perturbs B-cell homeostasis thus adding a new layer of immune dysregulation concurring to the increased susceptibility to develop autoimmunity in WAS patients.},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid23845947,

title = {Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome},

year  = {2013},

date = {2013-01-01},

journal = {Science},

volume = {341},

number = {6148},

pages = {1233151},

keywords = {},

pubstate = {published},

tppubtype = {article}

}