Canavan disease (CD) is a debilitating and lethal leukodystrophy caused by mutations in the aspartoacylase (
Dominic J. Gessler, Danning Li, Hongxia Xu, Qin Su, Julio Sanmiguel, Serafettin Tuncer, Constance Moore, Jean King, Reuben Matalon, Guangping Gao
Dose reduction of 3rd generation therapy achieves efficacious disease rescue in Canavan disease knockout (CD KO) mice.