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351 results

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Page 1
In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.
Biasco L, Pellin D, Scala S, Dionisio F, Basso-Ricci L, Leonardelli L, Scaramuzza S, Baricordi C, Ferrua F, Cicalese MP, Giannelli S, Neduva V, Dow DJ, Schmidt M, Von Kalle C, Roncarolo MG, Ciceri F, Vicard P, Wit E, Di Serio C, Naldini L, Aiuti A. Biasco L, et al. Among authors: roncarolo mg. Cell Stem Cell. 2016 Jul 7;19(1):107-19. doi: 10.1016/j.stem.2016.04.016. Epub 2016 May 26. Cell Stem Cell. 2016. PMID: 27237736 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.
In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells.
Biasco L, Scala S, Basso Ricci L, Dionisio F, Baricordi C, Calabria A, Giannelli S, Cieri N, Barzaghi F, Pajno R, Al-Mousa H, Scarselli A, Cancrini C, Bordignon C, Roncarolo MG, Montini E, Bonini C, Aiuti A. Biasco L, et al. Among authors: roncarolo mg. Sci Transl Med. 2015 Feb 4;7(273):273ra13. doi: 10.1126/scitranslmed.3010314. Sci Transl Med. 2015. PMID: 25653219
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.
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.
Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C. Aiuti A, et al. Among authors: roncarolo mg. J Clin Invest. 2007 Aug;117(8):2233-40. doi: 10.1172/JCI31666. J Clin Invest. 2007. PMID: 17671653 Free PMC article. Clinical Trial.
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.
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.
Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell.
Biasco L, Ambrosi A, Pellin D, Bartholomae C, Brigida I, Roncarolo MG, Di Serio C, von Kalle C, Schmidt M, Aiuti A. Biasco L, et al. Among authors: roncarolo mg. EMBO Mol Med. 2011 Feb;3(2):89-101. doi: 10.1002/emmm.201000108. Epub 2011 Jan 17. EMBO Mol Med. 2011. PMID: 21243617 Free PMC article. Clinical Trial.
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L. Biffi A, et al. Among authors: roncarolo mg. Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11. Science. 2013. PMID: 23845948 Clinical Trial.
351 results