Aiuti A - Search Results

1

Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs.

Crippa S, Conti A, Vavassori V, Ferrari S, Beretta S, Rivis S, Bosotti R, Scala S, Pirroni S, Jofra-Hernandez R, Santi L, Basso-Ricci L, Merelli I, Genovese P, Aiuti A, Naldini L, Di Micco R, Bernardo ME. Crippa S, et al. Among authors: aiuti a. Mol Ther. 2023 Jan 4;31(1):230-248. doi: 10.1016/j.ymthe.2022.08.011. Epub 2022 Aug 17. Mol Ther. 2023. PMID: 35982622 Free PMC article.

2

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: aiuti a. Hum Gene Ther. 2001 Nov 1;12(16):1979-88. doi: 10.1089/104303401753204553. Hum Gene Ther. 2001. PMID: 11686939

3

Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.

Dupré L, Trifari S, Follenzi A, Marangoni F, Lain de Lera T, Bernad A, Martino S, Tsuchiya S, Bordignon C, Naldini L, Aiuti A, Roncarolo MG. Dupré L, et al. Among authors: aiuti a. Mol Ther. 2004 Nov;10(5):903-15. doi: 10.1016/j.ymthe.2004.08.008. Mol Ther. 2004. PMID: 15509508 Free article.

4

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: aiuti a. Mol Ther. 2004 Dec;10(6):1096-108. doi: 10.1016/j.ymthe.2004.08.014. Mol Ther. 2004. PMID: 15564141 Free article.

5

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: aiuti a. Hum Gene Ther. 2006 Mar;17(3):303-13. doi: 10.1089/hum.2006.17.303. Hum Gene Ther. 2006. PMID: 16544979

6

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: aiuti a. 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.

7

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A. Cassani B, et al. Among authors: aiuti a. Blood. 2009 Oct 22;114(17):3546-56. doi: 10.1182/blood-2009-02-202085. Epub 2009 Aug 3. Blood. 2009. PMID: 19652199 Free article. Clinical Trial.

8

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: aiuti a. 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

9

Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome.

Catucci M, Prete F, Bosticardo M, Castiello MC, Draghici E, Locci M, Roncarolo MG, Aiuti A, Benvenuti F, Villa A. Catucci M, et al. Among authors: aiuti a. Gene Ther. 2012 Dec;19(12):1150-8. doi: 10.1038/gt.2011.202. Epub 2011 Dec 22. Gene Ther. 2012. PMID: 22189416 Free PMC article.

10

Retroviral integrations in gene therapy trials.

Biasco L, Baricordi C, Aiuti A. Biasco L, et al. Among authors: aiuti a. Mol Ther. 2012 Apr;20(4):709-16. doi: 10.1038/mt.2011.289. Epub 2012 Jan 17. Mol Ther. 2012. PMID: 22252453 Free PMC article. Review.

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