ASPA and Canavan disease: In a preclinical study for Canavan disease in adult Canavan mice, delivery of an AAV/Olig001-vector with oligodendroglial aspartoacylase (ASPA) cDNA through intracerebroventricular infusion into the cerebrospinal fluid (CSF) resulted in a dose-dependent rescue of ASPA activity, motor function, and a near-total reduction in vacuolation.