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Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A. Marangoni F, et al. Among authors: roncarolo mg. Mol Ther. 2009 Jun;17(6):1073-82. doi: 10.1038/mt.2009.31. Epub 2009 Mar 3. Mol Ther. 2009. PMID: 19259069 Free PMC article.
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. Aiuti A, et al. Among authors: roncarolo mg. Science. 2013 Aug 23;341(6148):1233151. doi: 10.1126/science.1233151. Epub 2013 Jul 11. Science. 2013. PMID: 23845947 Free PMC article. Clinical Trial.
The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function.
Locci M, Draghici E, Marangoni F, Bosticardo M, Catucci M, Aiuti A, Cancrini C, Marodi L, Espanol T, Bredius RG, Thrasher AJ, Schulz A, Litzman J, Roncarolo MG, Casorati G, Dellabona P, Villa A. Locci M, et al. Among authors: roncarolo mg. J Exp Med. 2009 Apr 13;206(4):735-42. doi: 10.1084/jem.20081773. Epub 2009 Mar 23. J Exp Med. 2009. PMID: 19307326 Free PMC article.
Gene therapy for primary immunodeficiency.
Booth C, Romano R, Roncarolo MG, Thrasher AJ. Booth C, et al. Among authors: roncarolo mg. Hum Mol Genet. 2019 Oct 1;28(R1):R15-R23. doi: 10.1093/hmg/ddz170. Hum Mol Genet. 2019. PMID: 31297531 Review.
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. Bosticardo M, et al. Among authors: roncarolo mg. J Allergy Clin Immunol. 2011 Jun;127(6):1376-84.e5. doi: 10.1016/j.jaci.2011.03.030. Epub 2011 Apr 29. J Allergy Clin Immunol. 2011. PMID: 21531013
Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models".
Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A. Marangoni F, et al. Among authors: roncarolo mg. Mol Ther. 2009 Jul;17(7):1300. doi: 10.1038/mt.2009.61. Epub 2016 Dec 6. Mol Ther. 2009. PMID: 28178476 Free PMC article. No abstract available.
350 results