ライフサイエンスコーパス: alpha-Gal A

1 alpha-Gal A activity in the organs became equal to or hi

2 alpha-Gal A infusions were well tolerated in all patient

3 esults from deficient alpha-galactosidase A (alpha-Gal A) activity and the pathologic accumulation of

4 -linked deficiency of alpha-galactosidase A (alpha-Gal A).

5 ked inherited loss of alpha-galactosidase A (alpha-Gal A).

6 activity of lysosomal alpha-galactosidase A (alpha-Gal A).

7 ed by a deficiency of alpha-galactosidase A (alpha-Gal A).

8 ed by a deficiency of alpha-galactosidase A (alpha-Gal A).

9 es upon inhibition of alpha-galactosidase A (alpha-Gal-A) in lysosomes.

10 ry disease (deficient alpha-galactosidase A [alpha-Gal A] activity) were performed in alpha-Gal A-def

11 ce of tissue and plasma GL-3 by administered alpha-Gal A, thereby providing the in vivo rationale-and

12 By 4 mo of age, alpha-Gal A -/0 mouse aortic endothelial cells achieved

13 ence with G, A, C, or T at nt 9331 within an alpha-Gal A complementary DNA expression vector were pre

14 Both GCase and alpha-Gal A are more stable at lysosomal pH with and wit

15 e results provide insight into how GCase and alpha-Gal A are thermodynamically stabilized by iminosug

16 ted the structure and stability of GCase and alpha-Gal A in a neutral-pH environment reflective of th

17 aortic endothelial cells from wild-type and alpha-Gal A -/0 mice were established.

18 patterns were compared between wild-type and alpha-Gal A null cells.

19 e synthase, restored cholesterol in cultured alpha-Gal A-deficient mouse aortic endothelial cell cave

20 revealed that the excessive Gb3 in cultured alpha-Gal A-deficient mouse aortic endothelial cells acc

21 Importantly, cells from alpha-Gal A -/0 mice but not alpha-Gal A +/0 mice expres

22 cultured embryonic fibroblasts derived from alpha-Gal A(-/0) mice were corrected by transducing thes

23 mouse aortic endothelial cells isolated from alpha-Gal A null mice was measured.

24 Thermodynamic parameters obtained from alpha-Gal A unfolding indicate two-state, van't Hoff unf

25 sidase (GCase) and acid-alpha-galactosidase (alpha-Gal A) hydrolyze the sphingolipids glucosyl- and g

26 Recently, we generated alpha-Gal A gene knockout mouse lines and described the

27 rug resistance retroviruses containing human alpha-Gal A cDNA.

28 Preclinical studies of recombinant human alpha-Gal A (r-halphaGalA) infusions in knockout mice de

29 s were determined for four recombinant human alpha-Gal A glycoforms, which differed in sialic acid an

30 Depleting Gb3 with recombinant human alpha-Gal A protein or d-threo-ethylenedioxyphenyl-P4, a

31 By contrast, recombinant human alpha-Gal A was less effective in normalizing the choles

32 - or DGJ-dependent conformational changes in alpha-Gal A are not seen.

33 globotriaosylceramide levels was observed in alpha-Gal A(-) males treated for 4 weeks with D-t-EtDO-P

34 A [alpha-Gal A] activity) were performed in alpha-Gal A-deficient mice.

35 tly, cells from alpha-Gal A -/0 mice but not alpha-Gal A +/0 mice expressed high levels of the globo-

36 Histopathological analysis of alpha-Gal A -/0 mice revealed subclinical lesions in the

37 The deficiency of alpha-Gal A activity and the accumulation of material co

38 een, and heart with concomitant elevation of alpha-Gal A activity.

39 We report here the generation of alpha-Gal A deficient mice by gene targeting and an anal

40 The knockout mice display a complete lack of alpha-Gal A activity.

41 ycin (DGJ), including the first structure of alpha-Gal A with DGJ.

42 Moreover, microbes induced inhibition of alpha-Gal-A activity in antigen-presenting cells.

43 present TLR-dependent negative regulation of alpha-Gal-A as a mechanistic link between pathogen recog

44 When 8-week-old alpha-Gal A(-) males were treated for 8 weeks with 10 mg

45 Recombinant alpha-Gal A more significantly lowered Gb3 levels by 48

46 Here, we show that alpha-Gal-A deficiency caused vigorous activation of NKT

47 57-nucleotide (nt) intronic sequence to the alpha-Gal A transcript from intron 4 of the gene has bee

48 our results with analogous experiments using alpha-Gal A and the chaperone 1-deoxygalactonijirimycin