Article Abstract

Brain N-acetylaspartate accumulation in Canavan disease is not neurotoxic per se: the implications of the first gene replacement therapy study to demonstrate successful post-symptomatic treatment in mice

Authors: Christina Elliott, Hussam Al-Humadi, Rodrigo Gutierrez-Quintana, Apostolos Zarros

Abstract

Canavan disease (CD) is identified as a fatal spongiform leukodystrophy caused by missense mutations in the Aspa gene (1,2).