Naldini L - Search Results

1

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.

Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME. Calbi V, et al. Among authors: naldini l. Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. Bone Marrow Transplant. 2018. PMID: 29379168 Free PMC article. No abstract available.

2

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice.

Consiglio A, Quattrini A, Martino S, Bensadoun JC, Dolcetta D, Trojani A, Benaglia G, Marchesini S, Cestari V, Oliverio A, Bordignon C, Naldini L. Consiglio A, et al. Among authors: naldini l. Nat Med. 2001 Mar;7(3):310-6. doi: 10.1038/85454. Nat Med. 2001. PMID: 11231629

3

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.

Biffi A, De Palma M, Quattrini A, Del Carro U, Amadio S, Visigalli I, Sessa M, Fasano S, Brambilla R, Marchesini S, Bordignon C, Naldini L. Biffi A, et al. Among authors: naldini l. J Clin Invest. 2004 Apr;113(8):1118-29. doi: 10.1172/JCI19205. J Clin Invest. 2004. PMID: 15085191 Free PMC article.

4

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: naldini l. Mol Ther. 2004 Nov;10(5):903-15. doi: 10.1016/j.ymthe.2004.08.008. Mol Ther. 2004. PMID: 15509508 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: naldini l. Hum Gene Ther. 2006 Mar;17(3):303-13. doi: 10.1089/hum.2006.17.303. Hum Gene Ther. 2006. PMID: 16544979

6

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.

Biffi A, Capotondo A, Fasano S, del Carro U, Marchesini S, Azuma H, Malaguti MC, Amadio S, Brambilla R, Grompe M, Bordignon C, Quattrini A, Naldini L. Biffi A, et al. Among authors: naldini l. J Clin Invest. 2006 Nov;116(11):3070-82. doi: 10.1172/JCI28873. J Clin Invest. 2006. PMID: 17080200 Free PMC article.

7

Novel candidate disease for gene therapy: metachromatic leukodystrophy.

Biffi A, Naldini L. Biffi A, et al. Among authors: naldini l. Expert Opin Biol Ther. 2007 Aug;7(8):1193-205. doi: 10.1517/14712598.7.8.1193. Expert Opin Biol Ther. 2007. PMID: 17696818 Review.

8

Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.

Capotondo A, Cesani M, Pepe S, Fasano S, Gregori S, Tononi L, Venneri MA, Brambilla R, Quattrini A, Ballabio A, Cosma MP, Naldini L, Biffi A. Capotondo A, et al. Among authors: naldini l. Hum Gene Ther. 2007 Sep;18(9):821-36. doi: 10.1089/hum.2007.048. Hum Gene Ther. 2007. PMID: 17845130

9

Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy.

Cesani M, Capotondo A, Plati T, Sergi LS, Fumagalli F, Roncarolo MG, Naldini L, Comi G, Sessa M, Biffi A. Cesani M, et al. Among authors: naldini l. Hum Mutat. 2009 Oct;30(10):E936-45. doi: 10.1002/humu.21093. Hum Mutat. 2009. PMID: 19606494

10

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: naldini l. 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.

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