• Corpus ID: 20475738

Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.

@article{Leone1999GlobalCG,
  title={Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease.},
  author={Paola Leone and Christopher G. Janson and Scott McPhee and Matthew John During},
  journal={Current opinion in molecular therapeutics},
  year={1999},
  volume={1 4},
  pages={
          487-92
        }
}
The neurogenetic prototypic disease on which we chose to test our gene therapy strategy is Canavan disease (CD). CD is an autosomal recessive leukodystrophy associated with spongiform degeneration of the brain. At present the disease is uniformly fatal in affected probands. CD is characterized by mutations in the aspartoacylase (ASPA) gene, resulting in loss of enzyme activity. In this review, recent evidence is summarized on the etiology and possible treatments for CD. In particular, we… 

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