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pubmed-article:10894262pubmed:abstractTextCanavan 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 that can be used for gene transfer.lld:pubmed
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pubmed-article:10894262pubmed:articleTitleKnock-out mouse for Canavan disease: a model for gene transfer to the central nervous system.lld:pubmed
pubmed-article:10894262pubmed:affiliationDepartment of Pediatrics, Children's Hospital, UTMB Galveston, TX 77555-0359, USA. rmatalon@utmb.edulld:pubmed
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