Department of Chemistry, International Institute for Nanotechnology, Northwestern University, Evanston, IL 60208, USA.
Department of Physiology and Biophysics, Case Western Reserve University School of Medicine, OH 44106, USA.
Sci Adv. 2024 Nov;10(44):eado8307. doi: 10.1126/sciadv.ado8307. Epub 2024 Nov 1.
Recently, it has been shown that blocking the binding of valosin-containing protein (VCP) to mutant huntingtin (mtHtt) can prevent neuronal mitochondrial autophagy in Huntington's disease (HD) models. Herein, we describe the development and efficacy of a protein-like polymer (PLP) for inhibiting this interaction in cellular and in vivo models of HD. PLPs exhibit bioactivity in HD mouse striatal cells by successfully inhibiting mitochondrial destruction. PLP is notably resilient to in vitro enzyme, serum, and liver microsome stability assays, which render analogous control oligopeptides ineffective. PLP demonstrates a 2000-fold increase in circulation half-life compared to peptides, exhibiting an elimination half-life of 152 hours. In vivo efficacy studies in HD transgenic mice (R6/2) confirm the superior bioactivity of PLP compared to free peptide through behavioral and neuropathological analyses. PLP functions by preventing pathologic VCP/mtHtt binding in HD animal models; exhibits enhanced efficacy over the parent, free peptide; and implicates the PLP as a platform with potential for translational central nervous system therapeutics.
最近的研究表明,阻断富含缬氨酸蛋白(VCP)与突变型亨廷顿蛋白(mtHtt)的结合可以防止亨廷顿病(HD)模型中的神经元线粒体自噬。在此,我们描述了一种蛋白样聚合物(PLP)的开发和功效,该聚合物可抑制 HD 细胞和体内模型中的这种相互作用。PLP 通过成功抑制线粒体破坏,在 HD 小鼠纹状体细胞中表现出生物活性。PLP 明显耐受体外酶、血清和肝微粒体稳定性测定,而类似的对照寡肽则无效。与肽相比,PLP 的循环半衰期增加了 2000 倍,消除半衰期为 152 小时。在 HD 转基因小鼠(R6/2)的体内疗效研究中,通过行为和神经病理学分析证实了 PLP 与游离肽相比具有更高的生物活性。PLP 通过防止 HD 动物模型中的病理性 VCP/mtHtt 结合发挥作用;与母体游离肽相比,表现出增强的功效;并暗示 PLP 作为一个具有转化中枢神经系统治疗潜力的平台。