Article
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
Blood - 9 Dec 2010
Visigalli Ilaria, Delai Stefania, Politi Letterio S, Di Domenico Carmela, Cerri Federica, Mrak Emanuela, D'Isa Raffaele, Ungaro Daniela, Stok Merel, Sanvito Francesca, Mariani Elisabetta, Staszewsky Lidia, Godi Claudia, Russo Ilaria, Cecere Francesca, Del Carro Ubaldo, Rubinacci Alessandro, Brambilla Riccardo, Quattrini Angelo, Di Natale Paola, Ponder Katherine, Naldini Luigi, Biffi Alessandra
Abstract excerpt
Type I mucopolysaccharidosis (MPS I) is a lysosomal storage disorder caused by the deficiency of α-L-iduronidase, which results in glycosaminoglycan accumulation in tissues. Clinical manifestations include skeletal dysplasia, joint stiffness, visual and auditory defects, cardiac insufficiency, he...
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