Delivery of genetic medicines for muscular dystrophies

被引：1

作者：

Chulanova, Yulia ^{[1
,2
,3
,4
]}

Breier, Dor ^{[1
,2
,3
,4
]}

Peer, Dan ^{[1
,2
,3
,4
]}

机构：

[1] Tel Aviv Univ, George S Wise Fac Life Sci, Shmunis Sch Biomed & Canc Res, Lab Precis Nanomed, Tel Aviv, Israel

[2] Tel Aviv Univ, Iby & Aladar Fleischman Fac Engn, Dept Mat Sci & Engn, Tel Aviv, Israel

[3] Tel Aviv Univ, Ctr Nanosci & Nanotechnol, Tel Aviv, Israel

[4] Tel Aviv Univ, Canc Biol Res Ctr, Tel Aviv, Israel

来源：

CELL REPORTS MEDICINE | 2025年 / 6卷 / 01期

关键词：

ADENOASSOCIATED VIRUS; SKELETAL-MUSCLE; OFF-TARGET; STABLE TRANSDUCTION; SATELLITE CELLS; MOUSE MODEL; MDX MICE; DUCHENNE; THERAPY; AAV;

D O I：

10.1016/j.xcrm.2024.101885

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

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.

引用

页数：15

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