Peraino Jaclyn, Zhang Huiping, Hermanrud Christina E, Li Guoying, Sachs David H, Huang Christene A, Wang Zhirui
Transplantation Biology Research Center, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
Protein Expr Purif. 2012 Apr;82(2):270-8. doi: 10.1016/j.pep.2012.01.012. Epub 2012 Feb 2.
Co-stimulation blockade can be used to modulate the immune response for induction of organ transplantation tolerance, treatment of autoimmune disease as well as cancer treatment. Cytotoxic T-Lymphocyte Antigen-4 (CTLA-4), also known as CD152, is an important co-stimulatory molecule which serves as a negative regulator for T cell proliferation and differentiation. CTLA-4/CD28-CD80/CD86 pathway is a critical co-stimulatory pathway for adaptive immune response. T cell activation through the T cell receptor and CD28 leads to increased expression of CTLA-4, an inhibitory receptor for CD80 and CD86. MGH MHC-defined miniature swine provide a unique large animal model useful for preclinical studies of transplantation tolerance and immune regulation. In this study, we have expressed the codon-optimized soluble porcine CTLA-4 in the yeast Pichia pastoris system. The secreted porcine CTLA-4 was captured using Ni-Sepharose 6 fast flow resin and further purified using strong anion exchange resin Poros 50HQ. Glycosylation analysis using PNGase F demonstrated the N-linked glycosylation on P. pastoris expressed soluble porcine CTLA-4. To improve the expression level and facilitate the downstream purification we mutated the two potential N-linked glycosylation sites with non-polarized alanines by site-directed mutagenesis. Removal of the two N-glycosylation sites significantly improved the production level from ∼2 to ∼8mg/L. Biotinylated glycosylated and non-N-glycosylated soluble porcine CTLA-4 both bind to a porcine CD80-expressing B-cell lymphoma cell line (K(D)=13nM) and competitively inhibit the binding of an anti-CD80 monoclonal antibody. The availability of soluble porcine CTLA-4, especially the non-N-glycosylated CTLA-4, will provide a very valuable tool for assessing co-stimulatory blockade treatment for translational studies in the clinically relevant porcine model.
共刺激阻断可用于调节免疫反应,以诱导器官移植耐受、治疗自身免疫性疾病以及用于癌症治疗。细胞毒性T淋巴细胞抗原4(CTLA-4),也被称为CD152,是一种重要的共刺激分子,作为T细胞增殖和分化的负调节因子。CTLA-4/CD28-CD80/CD86通路是适应性免疫反应的关键共刺激通路。通过T细胞受体和CD28激活T细胞会导致CTLA-4表达增加,CTLA-4是CD80和CD86的抑制性受体。MGH MHC定义的小型猪提供了一种独特的大型动物模型,可用于移植耐受和免疫调节的临床前研究。在本研究中,我们在酵母毕赤酵母系统中表达了密码子优化的可溶性猪CTLA-4。使用Ni-Sepharose 6快速流动树脂捕获分泌的猪CTLA-4,并使用强阴离子交换树脂Poros 50HQ进一步纯化。使用PNGase F进行的糖基化分析证明毕赤酵母表达的可溶性猪CTLA-4上存在N-连接糖基化。为了提高表达水平并便于下游纯化,我们通过定点诱变将两个潜在的N-连接糖基化位点突变为非极化丙氨酸。去除这两个N-糖基化位点显著提高了产量水平,从约2mg/L提高到约8mg/L。生物素化的糖基化和非N-糖基化可溶性猪CTLA-4均与表达猪CD80的B细胞淋巴瘤细胞系结合(解离常数K(D)=13nM),并竞争性抑制抗CD80单克隆抗体的结合。可溶性猪CTLA-4的可用性,尤其是非N-糖基化的CTLA-4,将为在临床相关猪模型中评估共刺激阻断治疗的转化研究提供非常有价值的工具。