Roncarolo MG - Search Results

1

Mobilized blood CD34+ cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice.

Deola S, Scaramuzza S, Birolo RS, Carballido-Perrig N, Ficara F, Mocchetti C, Dando J, Carballido JM, Bordignon C, Roncarolo MG, Bregni M, Aiuti A. Deola S, et al. Among authors: roncarolo mg. Hum Gene Ther. 2004 Mar;15(3):305-11. doi: 10.1089/104303404322886156. Hum Gene Ther. 2004. PMID: 15018739

2

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.

3

Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.

Dupré L, Marangoni F, Scaramuzza S, Trifari S, Hernández RJ, Aiuti A, Naldini L, Roncarolo MG. Dupré L, et al. Among authors: roncarolo mg. Hum Gene Ther. 2006 Mar;17(3):303-13. doi: 10.1089/hum.2006.17.303. Hum Gene Ther. 2006. PMID: 16544979

4

Efficient gene transfer into primitive hematopoietic progenitors using a bone marrow microenvironment cell line engineered to produce retroviral vectors.

Dando JS, Ficara F, Deola S, Roncarolo MG, Bordignon C, Aiuti A. Dando JS, et al. Among authors: roncarolo mg. Haematologica. 2004 Apr;89(4):462-70. Haematologica. 2004. PMID: 15075080

5

IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.

Ficara F, Superchi DB, Hernández RJ, Mocchetti C, Carballido-Perrig N, Andolfi G, Deola S, Colombo A, Bordignon C, Carballido JM, Roncarolo MG, Aiuti A. Ficara F, et al. Among authors: roncarolo mg. Mol Ther. 2004 Dec;10(6):1096-108. doi: 10.1016/j.ymthe.2004.08.014. Mol Ther. 2004. PMID: 15564141 Free article.

6

Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34+ cells.

Deola S, Scaramuzza S, Birolo RS, Cergnul M, Ficara F, Dando J, Voena C, Vai S, Monari M, Pogliani E, Corneo G, Peccatori J, Selleri S, Bordignon C, Roncarolo MG, Aiuti A, Bregni M. Deola S, et al. Among authors: roncarolo mg. J Transl Med. 2007 Jul 12;5:35. doi: 10.1186/1479-5876-5-35. J Transl Med. 2007. PMID: 17626627 Free PMC article.

7

A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors.

Dando JS, Roncarolo MG, Bordignon C, Aiuti A. Dando JS, et al. Among authors: roncarolo mg. Hum Gene Ther. 2001 Nov 1;12(16):1979-88. doi: 10.1089/104303401753204553. Hum Gene Ther. 2001. PMID: 11686939

8

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.

9

Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

Charrier S, Dupré L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O, Cattaneo F, Aiuti A, Eckenberg R, Thrasher AJ, Roncarolo MG, Galy A. Charrier S, et al. Among authors: roncarolo mg. Gene Ther. 2007 Mar;14(5):415-28. doi: 10.1038/sj.gt.3302863. Epub 2006 Oct 19. Gene Ther. 2007. PMID: 17051251

10

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.

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