Xia W, Ray W J, Ostaszewski B L, Rahmati T, Kimberly W T, Wolfe M S, Zhang J, Goate A M, Selkoe D J
Department of Neurology and Program in Neuroscience, Harvard Medical School and Center for Neurologic Diseases, Brigham and Women's Hospital, Boston, MA 02115, USA.
Proc Natl Acad Sci U S A. 2000 Aug 1;97(16):9299-304. doi: 10.1073/pnas.97.16.9299.
An unusual intramembranous cleavage of the beta-amyloid precursor protein (APP) by gamma-secretase is the final step in the generation of amyloid beta-peptide (Abeta). Two conserved aspartates in transmembrane (TM) domains 6 and 7 of presenilin (PS) 1 are required for Abeta production by gamma-secretase. Here we report that the APP C-terminal fragments, C83 and C99, which are the direct substrates of gamma-secretase, can be coimmunoprecipitated with both PS1 and PS2. PS/C83 complexes were detected in cells expressing endogenous levels of PS. The complexes accumulate when gamma-secretase is inactivated either pharmacologically or by mutating the PS aspartates. PS1/C83 and PS1/C99 complexes were detected in Golgi-rich and trans-Golgi network-rich vesicle fractions. In contrast, complexes of PS1 with APP holoprotein, which is not the immediate substrate of gamma-secretase, occurred earlier in endoplasmic reticulum-rich vesicles. The major portion of intracellular Abeta at steady state was found in the same Golgi/trans-Golgi network-rich vesicles, and Abeta levels in these fractions were markedly reduced when either PS1 TM aspartate was mutated to alanine. Furthermore, de novo generation of Abeta in a cell-free microsomal reaction occurred specifically in these same vesicle fractions and was markedly inhibited by mutating either TM aspartate. Thus, PSs are complexed with the gamma-secretase substrates C83 and C99 in the subcellular locations where Abeta is generated, indicating that PSs are directly involved in the pathogenically critical intramembranous proteolysis of APP.
γ-分泌酶对β-淀粉样前体蛋白(APP)进行的一种不寻常的膜内切割是生成β-淀粉样肽(Aβ)的最后一步。早老素(PS)1跨膜(TM)结构域6和7中的两个保守天冬氨酸是γ-分泌酶产生Aβ所必需的。在此我们报告,作为γ-分泌酶直接底物的APP C末端片段C83和C99可与PS1和PS2共同免疫沉淀。在表达内源性水平PS的细胞中检测到PS/C83复合物。当γ-分泌酶通过药理学方法失活或通过突变PS天冬氨酸而失活时,这些复合物会积累。在富含高尔基体和反式高尔基体网络的囊泡组分中检测到PS1/C83和PS1/C99复合物。相比之下,PS1与APP全蛋白(它不是γ-分泌酶的直接底物)的复合物在富含内质网的囊泡中更早出现。在稳态时,细胞内Aβ的主要部分存在于相同的富含高尔基体/反式高尔基体网络的囊泡中,当PS1的任何一个TM天冬氨酸突变为丙氨酸时,这些组分中的Aβ水平会显著降低。此外,在无细胞微粒体反应中从头生成Aβ特异性地发生在这些相同的囊泡组分中,并且通过突变任何一个TM天冬氨酸都能显著抑制。因此,PS在产生Aβ的亚细胞位置与γ-分泌酶底物C83和C99形成复合物,表明PS直接参与了APP具有致病关键意义的膜内蛋白水解过程。