Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.
Roca C, Motas S, Marcó S, Ribera A, Sánchez V, Sánchez X, Bertolin J, León X, Pérez J, Garcia M, et al. Hum Mol Genet. 2017 Apr 15; 26(8):1535-1551.