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Items: 23

1.

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

Sereni L, Castiello MC, Di Silvestre D, Della Valle P, Brombin C, Ferrua F, Cicalese MP, Pozzi L, Migliavacca M, Bernardo ME, Pignata C, Farah R, Notarangelo LD, Marcus N, Cattaneo L, Spinelli M, Giannelli S, Bosticardo M, van Rossem K, D'Angelo A, Aiuti A, Mauri P, Villa A.

J Allergy Clin Immunol. 2019 Sep;144(3):825-838. doi: 10.1016/j.jaci.2019.03.012. Epub 2019 Mar 27.

2.

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.

J Allergy Clin Immunol. 2018 Nov;142(5):1605-1617.e4. doi: 10.1016/j.jaci.2017.11.063. Epub 2018 Feb 13.

3.

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

Sereni L, Castiello MC, Marangoni F, Anselmo A, di Silvestre D, Motta S, Draghici E, Mantero S, Thrasher AJ, Giliani S, Aiuti A, Mauri P, Notarangelo LD, Bosticardo M, Villa A.

J Allergy Clin Immunol. 2018 Oct;142(4):1272-1284. doi: 10.1016/j.jaci.2017.12.1000. Epub 2018 Feb 6.

4.

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

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

J Allergy Clin Immunol. 2018 Sep;142(3):928-941.e8. doi: 10.1016/j.jaci.2017.11.015. Epub 2017 Dec 11.

5.

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.

Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F, Gennery AR, Bovolenta C, Palchaudhuri R, Scadden DT, Holmes MC, Villa A, Sitia G, Lombardo A, Genovese P, Naldini L.

Sci Transl Med. 2017 Oct 11;9(411). pii: eaan0820. doi: 10.1126/scitranslmed.aan0820.

PMID:
29021165
6.

Platelets in Wiskott-Aldrich syndrome: Victims or executioners?

Sereni L, Castiello MC, Villa A.

J Leukoc Biol. 2018 Mar;103(3):577-590. doi: 10.1189/jlb.5MR0617-257R. Epub 2017 Dec 29. Review.

PMID:
28851742
7.

In Vivo Chronic Stimulation Unveils Autoreactive Potential of Wiskott-Aldrich Syndrome Protein-Deficient B Cells.

Castiello MC, Pala F, Sereni L, Draghici E, Inverso D, Sauer AV, Schena F, Fontana E, Radaelli E, Uva P, Cervantes-Luevano KE, Benvenuti F, Poliani PL, Iannacone M, Traggiai E, Villa A, Bosticardo M.

Front Immunol. 2017 May 2;8:490. doi: 10.3389/fimmu.2017.00490. eCollection 2017.

8.

Intestinal microbiota sustains inflammation and autoimmunity induced by hypomorphic RAG defects.

Rigoni R, Fontana E, Guglielmetti S, Fosso B, D'Erchia AM, Maina V, Taverniti V, Castiello MC, Mantero S, Pacchiana G, Musio S, Pedotti R, Selmi C, Mora JR, Pesole G, Vezzoni P, Poliani PL, Grassi F, Villa A, Cassani B.

J Exp Med. 2016 Mar 7;213(3):355-75. doi: 10.1084/jem.20151116. Epub 2016 Feb 29.

9.

Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients.

Pala F, Morbach H, Castiello MC, Schickel JN, Scaramuzza S, Chamberlain N, Cassani B, Glauzy S, Romberg N, Candotti F, Aiuti A, Bosticardo M, Villa A, Meffre E.

J Clin Invest. 2015 Oct 1;125(10):3941-51. doi: 10.1172/JCI82249. Epub 2015 Sep 14.

10.

IL-10 critically modulates B cell responsiveness in Rankl-/- mice.

Marrella V, Lo Iacono N, Fontana E, Sobacchi C, Sic H, Schena F, Sereni L, Castiello MC, Poliani PL, Vezzoni P, Cassani B, Traggiai E, Villa A.

J Immunol. 2015 May 1;194(9):4144-53. doi: 10.4049/jimmunol.1401977. Epub 2015 Mar 30.

11.

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

Castiello MC, Scaramuzza S, Pala F, Ferrua F, Uva P, Brigida I, Sereni L, van der Burg M, Ottaviano G, Albert MH, Grazia Roncarolo M, Naldini L, Aiuti A, Villa A, Bosticardo M.

J Allergy Clin Immunol. 2015 Sep;136(3):692-702.e2. doi: 10.1016/j.jaci.2015.01.035. Epub 2015 Mar 16.

12.

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

Brigida I, Sauer AV, Ferrua F, Giannelli S, Scaramuzza S, Pistoia V, Castiello MC, Barendregt BH, Cicalese MP, Casiraghi M, Brombin C, Puck J, Müller K, Notarangelo LD, Montin D, van Montfrans JM, Roncarolo MG, Traggiai E, van Dongen JJ, van der Burg M, Aiuti A.

J Allergy Clin Immunol. 2014 Mar;133(3):799-806.e10. doi: 10.1016/j.jaci.2013.12.1043. Epub 2014 Feb 5.

13.

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

Castiello MC, Bosticardo M, Pala F, Catucci M, Chamberlain N, van Zelm MC, Driessen GJ, Pac M, Bernatowska E, Scaramuzza S, Aiuti A, Sauer AV, Traggiai E, Meffre E, Villa A, van der Burg M.

J Autoimmun. 2014 May;50:42-50. doi: 10.1016/j.jaut.2013.10.006. Epub 2013 Dec 25.

14.

Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity.

Catucci M, Zanoni I, Draghici E, Bosticardo M, Castiello MC, Venturini M, Cesana D, Montini E, Ponzoni M, Granucci F, Villa A.

Eur J Immunol. 2014 Apr;44(4):1039-45. doi: 10.1002/eji.201343935. Epub 2014 Jan 13.

15.

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

Aiuti A, Biasco L, Scaramuzza S, Ferrua F, Cicalese MP, Baricordi C, Dionisio F, Calabria A, Giannelli S, Castiello MC, Bosticardo M, Evangelio C, Assanelli A, Casiraghi M, Di Nunzio S, Callegaro L, Benati C, Rizzardi P, Pellin D, Di Serio C, Schmidt M, Von Kalle C, Gardner J, Mehta N, Neduva V, Dow DJ, Galy A, Miniero R, Finocchi A, Metin A, Banerjee PP, Orange JS, Galimberti S, Valsecchi MG, Biffi A, Montini E, Villa A, Ciceri F, Roncarolo MG, Naldini L.

Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11.

16.

Wiskott-Aldrich syndrome protein-mediated actin dynamics control type-I interferon production in plasmacytoid dendritic cells.

Prete F, Catucci M, Labrada M, Gobessi S, Castiello MC, Bonomi E, Aiuti A, Vermi W, Cancrini C, Metin A, Hambleton S, Bredius R, Notarangelo LD, van der Burg M, Kalinke U, Villa A, Benvenuti F.

J Exp Med. 2013 Feb 11;210(2):355-74. doi: 10.1084/jem.20120363. Epub 2013 Jan 21.

17.

SOCS1 gene transfer accelerates the transition to heart failure through the inhibition of the gp130/JAK/STAT pathway.

Cittadini A, Monti MG, Iaccarino G, Castiello MC, Baldi A, Bossone E, Longobardi S, Marra AM, Petrillo V, Saldamarco L, During MJ, Saccà L, Condorelli G.

Cardiovasc Res. 2012 Dec 1;96(3):381-90. doi: 10.1093/cvr/cvs261. Epub 2012 Aug 8.

18.

Autoimmunity in wiskott-Aldrich syndrome: an unsolved enigma.

Catucci M, Castiello MC, Pala F, Bosticardo M, Villa A.

Front Immunol. 2012 Jul 18;3:209. doi: 10.3389/fimmu.2012.00209. eCollection 2012.

19.

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome.

Scaramuzza S, Biasco L, Ripamonti A, Castiello MC, Loperfido M, Draghici E, Hernandez RJ, Benedicenti F, Radrizzani M, Salomoni M, Ranzani M, Bartholomae CC, Vicenzi E, Finocchi A, Bredius R, Bosticardo M, Schmidt M, von Kalle C, Montini E, Biffi A, Roncarolo MG, Naldini L, Villa A, Aiuti A.

Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Erratum in: Mol Ther. 2016 Jun;24(6):1159.

20.

Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome.

Catucci M, Prete F, Bosticardo M, Castiello MC, Draghici E, Locci M, Roncarolo MG, Aiuti A, Benvenuti F, Villa A.

Gene Ther. 2012 Dec;19(12):1150-8. doi: 10.1038/gt.2011.202. Epub 2011 Dec 22.

21.

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome.

Bosticardo M, Draghici E, Schena F, Sauer AV, Fontana E, Castiello MC, Catucci M, Locci M, Naldini L, Aiuti A, Roncarolo MG, Poliani PL, Traggiai E, Villa A.

J Allergy Clin Immunol. 2011 Jun;127(6):1376-84.e5. doi: 10.1016/j.jaci.2011.03.030. Epub 2011 Apr 29.

PMID:
21531013
22.

Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organs.

Trifari S, Scaramuzza S, Catucci M, Ponzoni M, Mollica L, Chiesa R, Cattaneo F, Lafouresse F, Calvez R, Vermi W, Medicina D, Castiello MC, Marangoni F, Bosticardo M, Doglioni C, Caniglia M, Aiuti A, Villa A, Roncarolo MG, Dupré L.

J Allergy Clin Immunol. 2010 Feb;125(2):439-448.e8. doi: 10.1016/j.jaci.2009.11.034.

PMID:
20159256
23.

Insulin-like growth factor-1 protects from vascular stenosis and accelerates re-endothelialization in a rat model of carotid artery injury.

Cittadini A, Monti MG, Castiello MC, D'Arco E, Galasso G, Sorriento D, Saldamarco L, De Paulis A, Napoli R, Iaccarino G, Saccà L.

J Thromb Haemost. 2009 Nov;7(11):1920-8. doi: 10.1111/j.1538-7836.2009.03607.x. Epub 2009 Sep 9.

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