INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris-Saclay, Evry, France.
Adv Exp Med Biol. 2021;1286:213-223. doi: 10.1007/978-3-030-55035-6_15.
Age-related neurodegenerative diseases have detrimental consequences on health of many patients and result in mortality. The current treatment options are limited and usually fail to correct the underlying pathology. AAV-based gene therapies have proved to be safe based on the data available on clinical trials for several monogenic diseases. Therefore, such therapies can pave the way to treat neurodegenerative diseases likes Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS). Here, the advantages of AAV-based gene therapies are discussed with emphasis on efforts of developing novel capsids with superior therapeutic efficacy. Furthermore, the results of clinical trials on AD, PD, and ALS are summarized.
与年龄相关的神经退行性疾病对许多患者的健康造成了不利影响，并导致死亡。目前的治疗选择有限，通常无法纠正潜在的病理。基于针对几种单基因疾病的临床试验中的可用数据，基于 AAV 的基因疗法已被证明是安全的。因此，此类疗法可以为治疗神经退行性疾病（如阿尔茨海默病（AD）、帕金森病（PD）和肌萎缩侧索硬化症（ALS））铺平道路。在这里，讨论了基于 AAV 的基因疗法的优点，并重点介绍了开发具有更好治疗效果的新型衣壳的努力。此外，还总结了 AD、PD 和 ALS 的临床试验结果。
