用于治疗肌肉萎缩症的基因药物递送

Delivery of genetic medicines for muscular dystrophies.

作者信息

Chulanova Yulia, Breier Dor, Peer Dan

机构信息

Laboratory of Precision Nanomedicine, The Shmunis School of Biomedicine and Cancer Research, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel-Aviv, Israel; Department of Materials Sciences and Engineering, Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel; Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel; Cancer Biology Research Center, Tel Aviv University, Tel Aviv, Israel.

出版信息

Cell Rep Med. 2025 Jan 21;6(1):101885. doi: 10.1016/j.xcrm.2024.101885. Epub 2025 Jan 6.

DOI:10.1016/j.xcrm.2024.101885

PMID:39765231

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11866442/

Abstract

Muscular dystrophies are a group of heterogenic disorders characterized by progressive muscle weakness, the most common of them being Duchenne muscular dystrophy (DMD). Muscular dystrophies are caused by mutations in over 50 distinct genes, and many of them are caused by different genetic mechanisms. Currently, none of these diseases have a cure. However, in recent years, significant progress has been made to correct the underlying genetic cause. The clinical development of adeno-associated viral vector-based therapies has simultaneously produced excitement and disappointment in the research community due to the moderate effect, making it clear that new methods of muscle delivery have to be created. Herein, we review the main characteristics of major muscular dystrophies and outline various muscle-targeted delivery methods being explored for genetic medicines.

摘要

肌肉萎缩症是一组以进行性肌肉无力为特征的异质性疾病，其中最常见的是杜氏肌营养不良症（DMD）。肌肉萎缩症由50多个不同基因的突变引起，其中许多是由不同的遗传机制导致的。目前，这些疾病均无法治愈。然而，近年来在纠正潜在遗传病因方面取得了重大进展。基于腺相关病毒载体的疗法的临床开发在研究界同时引起了兴奋和失望，因为其效果一般，这表明必须创造新的肌肉给药方法。在此，我们综述了主要肌肉萎缩症的主要特征，并概述了正在探索的用于基因药物的各种肌肉靶向给药方法。