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用于帕金森病免疫治疗的重组抗体片段。

Recombinant Antibody Fragments for Immunotherapy of Parkinson's Disease.

机构信息

Instituto de Investigaciones Biomedicas, Universidad Nacional Autonoma de Mexico (UNAM), Apartado Postal 70228, Cuidad Universitaria, CP 04510, Mexico, DF, Mexico.

出版信息

BioDrugs. 2024 Mar;38(2):249-257. doi: 10.1007/s40259-024-00646-5. Epub 2024 Jan 27.

Abstract

Parkinson's disease (PD) is the second most common age-related neurodegenerative disorder. Multiple genetic and environmental factors leading to progressive loss of dopaminergic neurons in the substantia nigra pars compacta (SN) and consequent depletion of dopamine were described. Current clinical approaches, such as dopamine replacement or deep brain stimulation using surgically implanted probes, provide symptomatic relief but cannot modify disease progression. Therefore, disease-modifying therapeutic tools are urgently needed. Immunotherapy approaches, including passive transfer of protective antibodies and their fragments, have shown therapeutic efficacy in several animal models of neurodegenerative diseases, including PD. Recombinant antibody fragments are promising alternatives to conventional full-length antibodies. Modern computational approaches and molecular biology tools, directed evolution methodology, and the design of tissue-penetrating fusion peptides allowed for the development of recombinant antibody fragments with superior specificity and affinity, reduced immunogenicity, the capacity to target hidden epitopes and cross the blood-brain barrier (BBB), higher solubility and stability, the ability to refold after heat denaturation, and inexpensive large-scale production. In addition, antibody fragments do not induce microglia Fcγ receptor (FcγR)-mediated proinflammatory response and tissue damage in the central nervous system (CNS), because they lack the Fc portion of the immunoglobulin molecule. In the present review, we summarized data on recombinant antibody fragments evaluated as immunotherapeutics in preclinical models of PD and discussed their potential for developing therapeutic and preventive protocols for patients with PD.

摘要

帕金森病(PD)是第二常见的与年龄相关的神经退行性疾病。描述了导致黑质致密部(SN)中多巴胺能神经元进行性丧失和多巴胺耗竭的多种遗传和环境因素。目前的临床方法,如使用手术植入探针的多巴胺替代或深部脑刺激,提供了症状缓解,但不能改变疾病进展。因此,迫切需要疾病修饰治疗工具。免疫疗法方法,包括保护性抗体及其片段的被动转移,已在包括 PD 在内的几种神经退行性疾病的动物模型中显示出治疗效果。重组抗体片段是传统全长抗体的有前途的替代品。现代计算方法和分子生物学工具、定向进化方法和穿透组织的融合肽设计允许开发具有更高特异性和亲和力、降低免疫原性、靶向隐藏表位和穿透血脑屏障(BBB)、更高溶解度和稳定性、能够在热变性后重新折叠以及廉价的大规模生产的重组抗体片段。此外,抗体片段不会在中枢神经系统(CNS)中诱导小胶质细胞 Fcγ 受体(FcγR)介导的促炎反应和组织损伤,因为它们缺乏免疫球蛋白分子的 Fc 部分。在本综述中,我们总结了评估作为 PD 临床前模型中免疫疗法的重组抗体片段的数据,并讨论了它们为 PD 患者开发治疗和预防方案的潜力。

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