Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. - Langford-Smith A; Wilkinson FL; Langford-Smith KJ; Holley RJ; Sergijenko A; Howe SJ; Bennett WR; Jones SA; Wraith J; Merry CL; Wynn RF; Bigger BW (Molecular therapy : the journal of the American Society of Gene Therapy, 2012)