Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
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L. Naldini | R. Brambilla | A. Biffi | K. Ponder | F. Sanvito | I. Visigalli | A. Quattrini | L. Politi | A. Rubinacci | C. Godi | L. Staszewsky | D. Ungaro | F. Cerri | Ilaria Russo | U. Del Carro | C. Di Domenico | P. di Natale | Raffaele d’Isa | Francesca Cecere | E. Mrak | M. Stok | E. Mariani | Stefania Delai
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