Skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA for Duchenne muscular dystrophy.

待确认 10 由 sailor 发布于 2026/6/18 9:48:17

DOI:10.1038/s41551-026-01689-5
作者：Yu Tian,?Yutong Liu,?Yuhao Tong,?Kristin Huntoon,?Liqun Yang,?Junfeng Shi,?Yifan Ma,?Shiyan Dong,?Seong Dong Jeong,?Andreanne Poppy Estania,?You Yi,?Kwang Joo Kwak,?Yifan Wang,?Annette Wu,?Jared L Edw
文献类型：期刊论文
补充材料：只需要正文
文献链接：https://doi.org/10.1038/s41551-026-01689-5

Nature

Abstract：Duchenne muscular dystrophy (DMD) is a severe, progressive muscle-wasting disorder caused by mutations in the DMD gene, which encodes dystrophin. Although gene therapy using viral vectors ha

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