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Page 1
Ten years of gene therapy for primary immune deficiencies.
Aiuti A, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. Hematology Am Soc Hematol Educ Program. 2009:682-9. doi: 10.1182/asheducation-2009.1.682. Hematology Am Soc Hematol Educ Program. 2009. PMID: 20008254 Review.
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. Scaramuzza S, et al. Among authors: roncarolo mg. Mol Ther. 2013 Jan;21(1):175-84. doi: 10.1038/mt.2012.23. Epub 2012 Feb 28. Mol Ther. 2013. PMID: 22371846 Free PMC article.
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817. N Engl J Med. 2009. PMID: 19179314 Free article. Clinical Trial.
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, Morecki S, Andolfi G, Tabucchi A, Carlucci F, Marinello E, Cattaneo F, Vai S, Servida P, Miniero R, Roncarolo MG, Bordignon C. Aiuti A, et al. Among authors: roncarolo mg. Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104. Science. 2002. PMID: 12089448 Clinical Trial.
Gene therapy for adenosine deaminase deficiency.
Aiuti A, Ficara F, Cattaneo F, Bordignon C, Roncarolo MG. Aiuti A, et al. Among authors: roncarolo mg. Curr Opin Allergy Clin Immunol. 2003 Dec;3(6):461-6. doi: 10.1097/00130832-200312000-00007. Curr Opin Allergy Clin Immunol. 2003. PMID: 14612670 Review.
Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects.
Mortellaro A, Hernandez RJ, Guerrini MM, Carlucci F, Tabucchi A, Ponzoni M, Sanvito F, Doglioni C, Di Serio C, Biasco L, Follenzi A, Naldini L, Bordignon C, Roncarolo MG, Aiuti A. Mortellaro A, et al. Among authors: roncarolo mg. Blood. 2006 Nov 1;108(9):2979-88. doi: 10.1182/blood-2006-05-023507. Epub 2006 Jul 11. Blood. 2006. PMID: 16835374 Free article.
350 results