Machen J, Bertera S, Chang Y, Bottino R, Balamurugan A N, Robbins P D, Trucco M, Giannoukakis N
Diabetes Institute, Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.
Gene Ther. 2004 Oct;11(20):1506-14. doi: 10.1038/sj.gt.3302320.
Islet transplantation is a viable long-term therapeutic alternative to daily insulin replacement for type I diabetes. The allogeneic nature of the transplants poses immunological challenges for routine clinical utility. Gene transfer of immunoregulatory molecules and those that improve insulin release kinetics provides rational approaches to facilitate allogeneic islet transplantation as a potential therapy. We have examined the efficacy of a soluble type 1 tumor necrosis factor receptor (TNFR) immunoglobulin-Fc fusion transgene (TNFR-Ig) to protect human islets from cytokine-induced apoptosis in culture, as well as in facilitating allogeneic islet transplants in diabetic mice. Cultured human islets were transduced with an adenoviral vector encoding human TNFR-Ig (Ad-TNFR-Ig). TNFR-Ig protein was secreted by cultured islets, as well as by transduced mouse islet transplants recovered from mouse recipients. Glucose-induced insulin release kinetics were comparable among untransduced, Ad-TNFR-Ig-infected human islets and vector-transduced islets exposed to cytokines. In parallel, Ad-TNFR-Ig-infected islets were protected from cytokine-induced apoptosis activation. Finally, diabetic mice transplanted with allogeneic islets expressing TNFR-Ig returned to and maintained normoglycemia significantly longer than untransduced islet recipients. These data support the potential utility of TNFR-Ig gene transfer to islets as a means of facilitating allogeneic islet transplantation.
胰岛移植是I型糖尿病患者长期替代每日胰岛素注射的一种可行的治疗方法。移植的异体性质给常规临床应用带来了免疫方面的挑战。免疫调节分子以及改善胰岛素释放动力学的分子的基因转移为促进异体胰岛移植成为一种潜在治疗方法提供了合理途径。我们研究了可溶性I型肿瘤坏死因子受体(TNFR)免疫球蛋白-Fc融合转基因(TNFR-Ig)在保护培养中的人胰岛免受细胞因子诱导的凋亡方面的功效,以及在促进糖尿病小鼠的异体胰岛移植方面的功效。用编码人TNFR-Ig的腺病毒载体转导培养的人胰岛(Ad-TNFR-Ig)。TNFR-Ig蛋白由培养的胰岛分泌,也由从小鼠受体中回收的转导的小鼠胰岛移植分泌。在未转导的、Ad-TNFR-Ig感染的人胰岛以及暴露于细胞因子的载体转导的胰岛中,葡萄糖诱导的胰岛素释放动力学相当。同时,Ad-TNFR-Ig感染的胰岛免受细胞因子诱导的凋亡激活。最后,移植了表达TNFR-Ig的异体胰岛的糖尿病小鼠恢复并维持正常血糖的时间明显长于未转导胰岛的受体。这些数据支持将TNFR-Ig基因转移到胰岛作为促进异体胰岛移植的一种手段的潜在效用。