Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.
Tessitore A, Faella A, O'Malley T, Cotugno G, Doria M, Kunieda T, Matarese G, Haskins M, Auricchio A.
Mol Ther. 2008 Jan;16(1):30-7.