靶向神经变性霍尔马克蛋白的适体。

IF 4 2区 医学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY Nucleic acid therapeutics Pub Date : 2022-04-22 DOI:10.1089/nat.2021.0091
Niloufar Mollasalehi, L. François-Moutal, David Porciani, D. H. Burke, M. Khanna
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引用次数: 2

摘要

神经变性是指神经结构的逐渐恶化,导致受影响患者的认知或运动障碍。对于阿尔茨海默氏症或帕金森氏症等最常见的神经退行性疾病,仍然没有有效的治疗方法。尽管NDs表现出不同的临床特征,但许多NDs的特征是在大脑和/或脊髓中积累错误折叠的蛋白质或肽片段。在患有不同NDs的患者中存在类似的纳入体,这为开发针对重叠疾病机制的疗法提供了理由。一种新的靶向策略涉及使用适体进行治疗开发。适体是能够识别具有高特异性和高亲和力的分子靶标的短核酸配体。尽管一些学术团体已经表明适体具有用于治疗和诊断应用的潜力,但它们的临床转化仍然有限。在这项研究中,我们描述了针对NDs相关蛋白开发的适体,包括朊病毒蛋白和淀粉样蛋白β(Aβ)、细胞表面受体和其他细胞质蛋白。这篇综述还描述了这些适体在成像、蛋白质检测和蛋白质定量方面的应用进展,并对其在疾病诊断和治疗中的加速临床应用提供了见解。
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Aptamers Targeting Hallmark Proteins of Neurodegeneration.
Neurodegeneration is a progressive deterioration of neural structures leading to cognitive or motor impairment of the affected patient. There is still no effective therapy for any of the most common neurodegenerative diseases (NDs) such as Alzheimer's or Parkinson's disease. Although NDs exhibit distinct clinical characteristics, many are characterized by the accumulation of misfolded proteins or peptide fragments in the brain and/or spinal cord. The presence of similar inclusion bodies in patients with diverse NDs provides a rationale for developing therapies directed at overlapping disease mechanisms. A novel targeting strategy involves the use of aptamers for therapeutic development. Aptamers are short nucleic acid ligands able to recognize molecular targets with high specificity and high affinity. Despite the fact that several academic groups have shown that aptamers have the potential to be used in therapeutic and diagnostic applications, their clinical translation is still limited. In this study, we describe aptamers that have been developed against proteins relevant to NDs, including prion protein and amyloid beta (Aβ), cell surface receptors and other cytoplasmic proteins. This review also describes advances in the application of these aptamers in imaging, protein detection, and protein quantification, and it provides insights about their accelerated clinical use for disease diagnosis and therapy.
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来源期刊
Nucleic acid therapeutics
Nucleic acid therapeutics BIOCHEMISTRY & MOLECULAR BIOLOGY-CHEMISTRY, MEDICINAL
CiteScore
7.60
自引率
7.50%
发文量
47
审稿时长
>12 weeks
期刊介绍: Nucleic Acid Therapeutics is the leading journal in its field focusing on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. The Journal examines many new approaches for using nucleic acids as therapeutic agents or in modifying nucleic acids for therapeutic purposes including: oligonucleotides, gene modification, aptamers, RNA nanoparticles, and ribozymes.
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