Article
Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system.
The journal of gene medicine - 1 Jan 2000
Matalon R, Rady P L, Platt K A, Skinner H B, Quast M J, Campbell G A, Matalon K, Ceci J D, Tyring S K, Nehls M, Surendran S, Wei J, Ezell E L, Szucs S
Abstract excerpt
BACKGROUND: Canavan disease (CD) is an autosomal recessive leukodystrophy characterized by deficiency of aspartoacylase (ASPA) and increased levels of N-acetylaspartic acid (NAA) in brain and body fluids, severe mental retardation and early death. Gene therapy has been attempted in a number of children with CD. The lack of an animal model has been a limiting factor in developing vectors for the treatment of CD....
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