Biffi A - Search Results

1

Therapeutic efficacy of intracerebral hematopoietic stem cell gene therapy in an Alzheimer's disease mouse model.

Milazzo R, Montepeloso A, Kumar R, Ferro F, Cavalca E, Rigoni P, Cabras P, Ciervo Y, Das S, Capotondo A, Pellin D, Peviani M, Biffi A. Milazzo R, et al. Among authors: biffi a. Nat Commun. 2024 Sep 13;15(1):8024. doi: 10.1038/s41467-024-52301-w. Nat Commun. 2024. PMID: 39271711 Free PMC article.

2

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. J Clin Invest. 2004 Apr;113(8):1118-29. doi: 10.1172/JCI19205. J Clin Invest. 2004. PMID: 15085191 Free PMC article.

3

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. J Clin Invest. 2006 Nov;116(11):3070-82. doi: 10.1172/JCI28873. J Clin Invest. 2006. PMID: 17080200 Free PMC article.

4

Human hematopoietic stem cells in gene therapy: pre-clinical and clinical issues.

Biffi A, Cesani M. Biffi A, et al. Curr Gene Ther. 2008 Apr;8(2):135-46. doi: 10.2174/156652308784049381. Curr Gene Ther. 2008. PMID: 18393833 Review.

5

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: biffi a. Hum Mutat. 2009 Oct;30(10):E936-45. doi: 10.1002/humu.21093. Hum Mutat. 2009. PMID: 19606494

6

Development and maturation of invariant NKT cells in the presence of lysosomal engulfment.

Plati T, Visigalli I, Capotondo A, Buono M, Naldini L, Cosma MP, Biffi A. Plati T, et al. Among authors: biffi a. Eur J Immunol. 2009 Oct;39(10):2748-54. doi: 10.1002/eji.200939639. Eur J Immunol. 2009. PMID: 19637231 Free article.

7

The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche.

Visigalli I, Ungari S, Martino S, Park H, Cesani M, Gentner B, Sergi Sergi L, Orlacchio A, Naldini L, Biffi A. Visigalli I, et al. Among authors: biffi a. Blood. 2010 Sep 16;116(11):1857-66. doi: 10.1182/blood-2009-12-256461. Epub 2010 May 28. Blood. 2010. PMID: 20511539 Free PMC article.

8

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

Visigalli I, Delai S, Politi LS, Di Domenico C, Cerri F, Mrak E, D'Isa R, Ungaro D, Stok M, Sanvito F, Mariani E, Staszewsky L, Godi C, Russo I, Cecere F, Del Carro U, Rubinacci A, Brambilla R, Quattrini A, Di Natale P, Ponder K, Naldini L, Biffi A. Visigalli I, et al. Among authors: biffi a. Blood. 2010 Dec 9;116(24):5130-9. doi: 10.1182/blood-2010-04-278234. Epub 2010 Sep 16. Blood. 2010. PMID: 20847202 Free PMC article.

9

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.

Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A, Schira G, Amendola M, Quattrini A, Martino S, Orlacchio A, Dick JE, Biffi A, Naldini L. Gentner B, et al. Among authors: biffi a. Sci Transl Med. 2010 Nov 17;2(58):58ra84. doi: 10.1126/scitranslmed.3001522. Sci Transl Med. 2010. PMID: 21084719

10

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. 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.

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