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1 jirimycin, was evaluated in a mouse model of Tay-Sachs disease.
2 ain at a level reported to be therapeutic in Tay-Sachs disease.
3 ng in the severe neurodegenerative disorder, Tay-Sachs disease.
4 osomal storage disorders, namely Gaucher and Tay-Sachs disease.
5 in tissues of an asymptomatic mouse model of Tay-Sachs disease, a severe human gangliosidosis, indica
6                                              Tay-Sachs disease, an inborn lysosomal disease featuring
7 erent lysosomal storage disorders, including Tay-Sachs disease and Gaucher disease, can be accounted
8 r deficiency (also known as the AB variant), Tay-Sachs disease, and Sandhoff disease are the major fo
9 an GM1 gangliosidosis, Sandhoff disease, and Tay-Sachs disease brains.
10                                        Thus, Tay-Sachs disease can be caused by the deficiency of eit
11  and leukocytes from patients with infantile Tay-Sachs disease, including a patient with thermolabile
12                                              Tay-Sachs disease is an inborn lysosomal disease charact
13 ects in GM2-AP result in an atypical form of Tay-Sachs disease known as variant AB GM2 gangliosidosis
14 e geographic distribution of the two primary Tay-Sachs disease mutations, with the first being more c
15  HEXA message in lymphoblasts derived from a Tay-Sachs disease patient homozygous for the common fram
16            Furthermore, as loss of GM2-AP in Tay-Sachs disease prevents degradation of GM2 gangliosid
17              The mouse model of human type B Tay-Sachs disease recently engineered by the targeted di
18 e, neurodegenerative conditions that include Tay-Sachs disease, Sandhoff disease, and the GM2 activat
19 ociated with the development of another LSD, Tay-Sachs disease, thus suggesting general applicability
20 he cystic fibrosis CFDelta508 allele and the Tay-Sachs disease TSD 1278 allele from single heterozygo
21                                              Tay-Sachs disease (TSD) is a classical glycosphingolipid
22                                              Tay-Sachs disease (TSD) is an autosomal recessive, neuro
23 (HexA) causes the lysosomal storage disorder Tay-Sachs disease (TSD).
24 d the observed results of the mouse model of Tay-Sachs disease, we have purified mouse liver Hex A an

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