Central nervous system diseases: innovative animal models from lab to clinic

Knock-out mouse for Canavan disease: a m

Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system

✍ Reuben Matalon; Peter L. Rady; Kenneth A. Platt; Henry B. Skinner; Michael J. Qu 📂 Article 📅 2000 🏛 John Wiley and Sons 🌐 English ⚖ 544 KB

Background Canavan disease (CD) is an autosomal recessive leukodystrophy characterized by de®ciency of aspartoacylase (ASPA) and increased levels of N-acetylaspartic acid (NAA) in brain and body ¯uids, severe mental retardation and early death. Gene therapy has been attempted in a number of children