Centro de Investigación Médica Aplicada (CIMA), Department of Neurosciences, Universidad de Navarra, 31008 Pamplona, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CiberNed), 28031 Madrid, Spain.
Int J Mol Sci. 2021 Jun 15;22(12):6389. doi: 10.3390/ijms22126389.
It is without any doubt that precision medicine therapeutic strategies targeting neurodegenerative disorders are currently witnessing the spectacular rise of newly designed approaches based on the use of viral vectors as Trojan horses for the controlled release of a given genetic payload. Among the different types of viral vectors, adeno-associated viruses (AAVs) rank as the ones most commonly used for the purposes of either disease modeling or for therapeutic strategies. Here, we reviewed the current literature dealing with the use of AAVs within the field of Parkinson's disease with the aim to provide neuroscientists with the advice and background required when facing a choice on which AAV might be best suited for addressing a given experimental challenge. Accordingly, here we will be summarizing some insights on different AAV serotypes, and which would be the most appropriate AAV delivery route. Next, the use of AAVs for modeling synucleinopathies is highlighted, providing potential readers with a landscape view of ongoing pre-clinical and clinical initiatives pushing forward AAV-based therapeutic approaches for Parkinson's disease and related synucleinopathies.
毫无疑问,目前针对神经退行性疾病的精准医学治疗策略正在见证基于使用病毒载体作为特洛伊木马来控制释放特定遗传有效载荷的新设计方法的惊人兴起。在不同类型的病毒载体中,腺相关病毒 (AAV) 是最常用于疾病建模或治疗策略的病毒载体。在这里,我们回顾了与帕金森病领域中使用 AAV 相关的当前文献,旨在为神经科学家在面对选择哪种 AAV 最适合解决特定实验挑战时提供所需的建议和背景。因此,在这里我们将总结一些关于不同 AAV 血清型的见解,以及哪种 AAV 传递途径最合适。接下来,强调了 AAV 用于模拟突触核蛋白病,为潜在读者提供了正在进行的基于 AAV 的治疗方法的临床前和临床计划的全景图,用于治疗帕金森病和相关的突触核蛋白病。