帕金森病:从遗传学和表观遗传学到治疗,一个基于miRNA的战略
Elena Paccosi1, Luca Proietti-De-Santis1
1Unit of Molecular Genetics of Aging, Department of Ecology and Biology (DEB), University of Tuscia, 01100 Viterbo, Italy.
International journal of molecular sciences
|June 10, 2023
概括
帕金森病 (PD) 涉及神经元和蛋白质聚合物的损失. 携带微RNA (miRNA) 的修饰外体提供了一个有前途的治疗策略,通过向表观遗传因素和克服血脑屏障.
科学领域:
- 神经科学是一个神经科学.
- 遗传学 是一个遗传学.
- 生物化学 生化学
背景情况:
- 帕金森病 (PD) 是一种常见的神经退行性疾病,其特征是多巴胺基神经元损失和勒维体形成.
- 目前PD的治疗方法提供症状缓解,但不能阻止疾病的进展,并可能导致副作用.
- 表观遗传失调,特别是涉及微RNAs (miRNAs),在PD病变发生过程中起着重要作用.
研究的目的:
- 提供对导致帕金森病的遗传和表观遗传因素的系统概述.
- 探索外体和miRNAs在治疗PD中的治疗潜力.
- 研究外体-miRNA网络用于新型PD疗法的临床应用.
主要方法:
- 对帕金森病的遗传和表观遗传基础的现有文献的审查.
- 分析microRNAs (miRNAs) 和外体在PD病原和潜在治疗中的作用.
- 检查介质干细胞 (MSC) 衍生出异体的治疗性输送.
主要成果:
- 有证据表明表观遗传变化,包括miRNA失调,对PD有显著的贡献.
- 修改后的外体可以被设计为通过血脑屏障传递治疗分子,包括RNA.
- 在临床前模型中,带有miRNA的介质干细胞 (MSC) 衍生外体显示了PD治疗的潜力.
结论:
- 外基因组-miRNA网络代表了开发帕金森病新型治疗策略的有希望的途径.
- 通过外体介导的miRNAs传递来向表观遗传机制提供了一种潜在的方法来克服PD进展.
- 对MSC衍生的外体和它们的miRNA载荷进行进一步的研究有必要在PD治疗中进行临床转化.
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