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344 results

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Page 1
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: von kalle c. Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11. Science. 2013. PMID: 23845948 Clinical Trial.
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection.
Biffi A, Bartolomae CC, Cesana D, Cartier N, Aubourg P, Ranzani M, Cesani M, Benedicenti F, Plati T, Rubagotti E, Merella S, Capotondo A, Sgualdino J, Zanetti G, von Kalle C, Schmidt M, Naldini L, Montini E. Biffi A, et al. Among authors: von kalle c. Blood. 2011 May 19;117(20):5332-9. doi: 10.1182/blood-2010-09-306761. Epub 2011 Mar 14. Blood. 2011. PMID: 21403130 Free 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: von kalle c. 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 genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.
Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M, Benedicenti F, Sergi LS, Ambrosi A, Ponzoni M, Doglioni C, Di Serio C, von Kalle C, Naldini L. Montini E, et al. Among authors: von kalle c. J Clin Invest. 2009 Apr;119(4):964-75. doi: 10.1172/JCI37630. Epub 2009 Mar 23. J Clin Invest. 2009. PMID: 19307726 Free PMC article.
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: von kalle c. 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.
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: von kalle c. 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.
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: von kalle c. 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 Vector Promoter is Decisive for Aberrant Transcript Formation.
Scholz SJ, Fronza R, Bartholomä CC, Cesana D, Montini E, von Kalle C, Gil-Farina I, Schmidt M. Scholz SJ, et al. Among authors: von kalle c. Hum Gene Ther. 2017 Oct;28(10):875-885. doi: 10.1089/hum.2017.162. Epub 2017 Aug 18. Hum Gene Ther. 2017. PMID: 28825370
344 results