Department of Pediatrics, Johns Hopkins School of Medicine, Baltimore, Maryland 21287, USA.
J Biol Chem. 2010 Apr 9;285(15):11314-25. doi: 10.1074/jbc.M109.044057. Epub 2010 Feb 10.
DeltaF508 cystic fibrosis transmembrane conductance regulator (CFTR) degradation involves ubiquitin modification and efficient proteasomal targeting of the nascent misfolded protein. We show that a deubiquitinating enzyme, ubiquitin C-terminal hydrolase-L1 (UCH-L1), is highly expressed in cystic fibrosis (CF) airway epithelial cells in vitro and in vivo. We hypothesized that the elevation in UCH-L1 in CF cells represents a cellular adaptation to counterbalance excessive proteasomal degradation. The bronchial epithelial cell lines IB3-1 (CF, high UCH-L1 expression) and S9 (non-CF, low UCH-L1 expression) were transiently transfected with wild type (WT) or DeltaF508 CFTR, WT UCH-L1 or small interfering RNA-UCH-L1, and a variety of ubiquitin mutants. We observed a positive correlation between UCH-L1 expression and steady state levels of WT- or DeltaF508-CFTR, and this stabilizing effect was confined to the early stages of CFTR synthesis. Immunolocalization of UCH-L1 by confocal microscopy revealed a partial co-localization with a ribosomal subunit and the endoplasmic reticulum. The UCH-L1-associated increase in CFTR levels was correlated with an increase in ubiquitinated CFTR (CFTR-Ub). Co-transfection with mutant ubiquitins and treatment with proteasome inhibitors suggested that UCH-L1 was reducing the proteasomal targeting of CFTR during synthesis by shortening conjugated polyubiquitin chains. Although not sufficient by itself to rescue mutant CFTR therapeutically, the elevation of UCH-L1 and its effect on CFTR processing provides insight into its potential roles in CF and other diseases.
DeltaF508 囊性纤维化跨膜电导调节因子 (CFTR) 的降解涉及泛素修饰和新生错误折叠蛋白的有效蛋白酶体靶向。我们表明,一种去泛素化酶,泛素 C 端水解酶-L1 (UCH-L1),在体外和体内的囊性纤维化 (CF) 气道上皮细胞中高度表达。我们假设 CF 细胞中 UCH-L1 的升高代表细胞适应以抵消过度的蛋白酶体降解。支气管上皮细胞系 IB3-1 (CF,高 UCH-L1 表达) 和 S9 (非 CF,低 UCH-L1 表达) 瞬时转染野生型 (WT) 或 DeltaF508 CFTR、WT UCH-L1 或小干扰 RNA-UCH-L1 以及各种泛素突变体。我们观察到 UCH-L1 表达与 WT 或 DeltaF508-CFTR 的稳态水平之间存在正相关,这种稳定作用仅限于 CFTR 合成的早期阶段。通过共聚焦显微镜对 UCH-L1 的免疫定位显示其与核糖体亚基和内质网有部分共定位。UCH-L1 相关的 CFTR 水平增加与泛素化 CFTR (CFTR-Ub) 的增加相关。共转染突变泛素和蛋白酶体抑制剂处理表明,UCH-L1 通过缩短连接的多泛素链来减少 CFTR 在合成过程中的蛋白酶体靶向。虽然本身不足以在治疗上拯救突变 CFTR,但 UCH-L1 的升高及其对 CFTR 加工的影响为其在 CF 和其他疾病中的潜在作用提供了深入了解。