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1 for the treatment of patients suffering from Canavan disease.
2 ion aspartoacylase mutations associated with Canavan Disease.
3 ng to preliminary studies in 2 children with Canavan disease.
4 iency of ASPA and should therefore result in Canavan disease.
5 nown patient of African-American origin with Canavan disease.
6 bral cortical thinning in an animal model of Canavan disease.
7 ith aspartoacylase that, when mutated, cause Canavan disease.
8 ial abnormalities such as Leighs disease and Canavans disease.
11 of the brain and is the established cause of Canavan disease, a fatal progressive leukodystrophy affe
13 tion of N-acetyl-l-aspartate, correlate with Canavan Disease, a neurodegenerative disorder usually fa
14 partate (NAA) is a characteristic feature of Canavan disease, a vacuolar leukodystrophy resulting fro
15 ve in vivo gene therapy for the treatment of Canavan disease and other neurological disorders, we dev
16 ral to the understanding the pathogenesis of Canavan disease and to the development of therapeutic st
18 ng Alzheimer's disease, Parkinson's disease, Canavan disease, aromatic l-amino acid decarboxylase [AA
20 ligodendrocytes, to deliver gene therapy for Canavan disease (CD), a rare leukodystrophy characterize
23 Aspartoacylase (ASPA)-deficient patients [Canavan disease (CD)] reportedly have increased urinary
24 , eight Gaucher mutations, four mutations in Canavan disease, four mutations in Fanconi anemia, and f
25 was aimed at finding the molecular basis of Canavan disease in 25 independent patients of non-Jewish
30 partoacylase (ASPA), the defective enzyme in Canavan disease, is detectable in the brain only in the
32 P183H, M195R, K213E/G274R, G274R, and F295S) Canavan Disease mutations resulted in undetectable enzym
34 neurodegenerative disease in 2 children with Canavan disease to assess the in vivo toxicity and effic
36 ration of NAA in the brains of children with Canavan disease would prevent or slow progression of neu