... Biochemistry and Medical Biotechnologies, University of Naples Federico II; Italy2Laboratory for Gene Transferand Therapy, Institute for Cancer Research and Treatment, University of Turin, ItalyMucopolysaccharidosis ... expression of the IDUA gene to cellsin vivo for treatment of MPS I.Keywords: MPS I; Hurler syndrome; a-L-iduronidase; gene therapy.Mucopolysaccharidosis type I (MPS I) is a lysosomaldisease due ... hematopoietic stem cell gene therapy for mucopolysaccharidosis type I. Gene Ther. 7,1875–1883.17. Hartung, S.D., Reddy, R.G., Whitley, C.B. & McIvor, R.S. (1999)Enzymatic correction and...