نتایج جستجو برای: canavan

تعداد نتایج: 211  

Journal: :Current opinion in molecular therapeutics 1999
P Leone C G Janson S J McPhee M J During

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, ...

2002
T R Olsen L Tranebjærg E A Kvittingen L Hagenfeldt C Møller Ø Nilssen

Canavan disease (CD) (OMIM 271900) is an autosomal recessive leucodystrophy characterised by swelling and spongy degeneration of the white matter of the brain. The biochemical marker for the disorder is increased level of N-acetylaspartic acid (NAA) in cerebrospinal fluid and urine owing to aspartoacylase (ASPA) deficiency. 2 The disease is caused by mutations in the gene encoding the enzyme as...

Journal: :Journal of medical genetics 2002
T R Olsen L Tranebjaerg E A Kvittingen L Hagenfeldt C Møller O Nilssen

Canavan disease (CD) (OMIM 271900) is an autosomal recessive leucodystrophy characterised by swelling and spongy degeneration of the white matter of the brain. The biochemical marker for the disorder is increased level of N-acetylaspartic acid (NAA) in cerebrospinal fluid and urine owing to aspartoacylase (ASPA) deficiency. 2 The disease is caused by mutations in the gene encoding the enzyme as...

Journal: :Clinical biochemistry 2005
Barbara Tavazzi Giuseppe Lazzarino Paola Leone Angela Maria Amorini Francesco Bellia Christopher G Janson Valentina Di Pietro Lia Ceccarelli Sonia Donzelli Jeremy S Francis Bruno Giardina

OBJECTIVES To set up a novel simple, sensitive, and reliable ion-pairing HPLC method for the synchronous separation of several purines, pyrimidines, N-acetylated amino acids, and dicarboxylic acids for the chemical diagnosis and screening of inborn errors of metabolism (IEM). DESIGN AND METHODS The separation was set up using a Hypersil C-18, 5-microm particle size, 250 x 4.6 mm column, and a...

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