Stem Cell Laboratory, Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, 20122 Milan, Italy.
Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.
Brain. 2023 Jun 1;146(6):2227-2240. doi: 10.1093/brain/awad014.
Neurodegenerative diseases are a major global health burden particularly with the increasing ageing population. Hereditary predisposition and environmental risk factors contribute to the heterogeneity of existing pathological phenotypes. Traditional clinical interventions focused on the use of small drugs have often led to failures due to the difficulties in crossing the blood-brain barrier and reaching the brain. In this regard, nanosystems can specifically deliver drugs and improve their bioavailability, overcoming some of the major challenges in neurodegenerative disease treatment. This review focuses on the use of nanosystems as an encouraging therapeutic approach targeting molecular pathways involved in localized and systematic neurodegenerative diseases. Among the latter, Friedreich's ataxia is an untreatable complex multisystemic disorder and the most widespread type of ataxia; it represents a test case to validate the clinical potential of therapeutic strategies based on nanoparticles with pleiotropic effects.
神经退行性疾病是全球主要的健康负担，尤其是随着人口老龄化的加剧。遗传易感性和环境风险因素导致了现有病理表型的异质性。传统的临床干预侧重于使用小分子药物，但由于血脑屏障的穿透性和到达大脑的难度，往往导致失败。在这方面，纳米系统可以专门递送药物并提高其生物利用度，克服神经退行性疾病治疗中的一些主要挑战。本综述重点介绍了纳米系统作为一种有前途的治疗方法，针对涉及局部和系统性神经退行性疾病的分子途径。在后者中，弗里德里希共济失调是一种无法治愈的复杂多系统疾病，也是最常见的共济失调类型；它是验证基于具有多效性的纳米颗粒的治疗策略的临床潜力的一个测试案例。
