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

@article{Matalon2000KnockoutMF,
  title={Knock-out mouse for Canavan disease: a model for gene transfer to the central nervous system.},
  author={Reuben Matalon and Peter L. Rady and Kathryn A. Platt and H. B. Skinner and Michael J. Quast and Gerald A. Campbell and Kim Matalon and Jeffrey D. Ceci and Stephen K Tyring and Michael Nehls and Sankar Surendran and Jiang Wei and E. L. Ezell and Sylvia Szucs},
  journal={The journal of gene medicine},
  year={2000},
  volume={2 3},
  pages={165-75}
}
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. This paper reports the successful creation of a knock-out mouse for Canavan disease… CONTINUE READING
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