基于载体传递的小干扰 RNA 治疗剂在治疗神经退行性疾病方面的进展。

Advances in carrier-delivered small interfering RNA based therapeutics for treatment of neurodegenerative diseases.

机构信息

Department of Neurology, Second Affiliated Hospital (Shanghai Changzheng Hospital) of Naval Medical University, Shanghai, China.

School of Health Science and Engineering, University of Shanghai for Science and Technology, Shanghai, China.

出版信息

Biomater Sci. 2024 Sep 25;12(19):4927-4945. doi: 10.1039/d4bm00878b.

DOI:10.1039/d4bm00878b

PMID:39206575

Abstract

Neurodegenerative diseases are devastating diseases that severely affect the health of people all over the world. RNA therapies have become one of the most promising critical drug treatments for neurodegenerative diseases due to their excellent gene and protein editing effects. However, the successful transport of RNA the systemic route to the central nervous system remains one of the major obstacles in treating neurodegenerative diseases. This review will focus on therapeutic RNA that can successfully overcome the blood-brain barrier (BBB), with particular attention to small interfering RNAs (siRNAs), focusing on different types of neurodegenerative disease treatment strategies and accelerating their translation into clinical practice.

摘要

神经退行性疾病是一种严重影响全球人类健康的破坏性疾病。由于 RNA 疗法具有出色的基因和蛋白质编辑效果，因此已成为治疗神经退行性疾病最有前途的关键药物治疗方法之一。然而，将 RNA 通过全身途径成功递送至中枢神经系统仍然是治疗神经退行性疾病的主要障碍之一。本综述将重点介绍能够成功克服血脑屏障（BBB）的治疗性 RNA，特别关注小干扰 RNA（siRNA），并侧重于不同类型的神经退行性疾病治疗策略，并加速将其转化为临床实践。