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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: delai s. 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.
Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.
Visigalli I, Delai S, Ferro F, Cecere F, Vezzoli M, Sanvito F, Chanut F, Benedicenti F, Spinozzi G, Wynn R, Calabria A, Naldini L, Montini E, Cristofori P, Biffi A. Visigalli I, et al. Among authors: delai s. Hum Gene Ther. 2016 Oct;27(10):813-829. doi: 10.1089/hum.2016.068. Hum Gene Ther. 2016. PMID: 27431943