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Lesch-Nyhan disease as a model for central nervous system directed gene therapy

โœ Scribed by Friedmann, Theodore

Book ID
112147004
Publisher
John Wiley and Sons
Year
1995
Tongue
English
Weight
891 KB
Volume
1
Category
Article
ISSN
1080-4013

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