The Therapeutic Potential of DNA Aptamer for Dialysis-Related Amyloidosis

Y. Motomiya
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Abstract

β2m is a precursor protein of the dialysis-related amyloidosis (DRA) and β2m lacking the N-terminal six residues of the mature protein (ΔN6β2m) is a one of amyloidogenic variant of β2m. In the previous study, we had showed an unfolding in the C-terminal of ΔN6β2m using the specific monoclonal antibody for ΔN6β2m. We had also reported firstly an aptamer specific for ΔN6β2m. ΔN6β2m-aptamers specifically and tightly bound to ΔN6β2m and inhibited fibril formation. These results suggest the potential of ΔN6β2m-aptamers as reagents for therapeutic tools to prevent amyloid deposits in dialysis patients.
DNA适体对透析相关淀粉样变性的治疗潜力
β2m是透析相关淀粉样变性(DRA)的前体蛋白,缺乏成熟蛋白n端6个残基的β2m (ΔN6β2m)是β2m的淀粉样变性之一。在之前的研究中,我们已经使用ΔN6β2m的特异性单克隆抗体在ΔN6β2m的c端展开。我们还首次报道了ΔN6β2m特异性适配体。ΔN6β2m-aptamers与ΔN6β2m紧密结合,抑制原纤维形成。这些结果表明ΔN6β2m-aptamers作为预防透析患者淀粉样蛋白沉积的治疗工具试剂的潜力。
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