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Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles
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Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles
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Journal Article
Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles
2025
Overview
Inefficient targeting of muscle stem cells (MuSCs), also called satellite cells, represents a major bottleneck of current therapeutic strategies for muscular dystrophies, as it precludes the possibility of promoting compensatory regeneration. Here we describe a muscle-targeting delivery platform, based on gold nanoparticles, that enables the release of therapeutic oligonucleotides into MuSCs. We demonstrate that AuNPs conjugation to an aptamer against α7/β1 integrin dimers directs either local or systemic delivery of microRNA-206 to MuSCs, thereby promoting muscle regeneration and improving muscle functionality, in a mouse model of Duchenne Muscular Dystrophy. We show here that this platform is biocompatible, non-toxic, and non-immunogenic, and it can be easily adapted for the release of a wide range of therapeutic oligonucleotides into diseased muscles.
Lack of muscle stem cell targeting limits the treatment of muscular dystrophies. Here, the authors describe a platform based on functionalized gold nanoparticles that allows selective delivery of therapeutic oligonucleotides and promotes skeletal muscle regeneration.
Publisher
Nature Publishing Group UK,Nature Publishing Group,Nature Portfolio
Subject
/ 13/100
/ 13/31
/ 13/51
/ 147/143
/ 38
/ 59
/ 64
/ 82/103
/ 82/58
/ Animals
/ Aptamers
/ Aptamers, Nucleotide - chemistry
/ Duchenne's muscular dystrophy
/ Gold
/ Humanities and Social Sciences
/ Humans
/ Metal Nanoparticles - chemistry
/ Mice
/ MicroRNAs - administration & dosage
/ miRNA
/ Muscle, Skeletal - physiology
/ Muscles
/ Muscular Dystrophy, Duchenne - genetics
/ Muscular Dystrophy, Duchenne - therapy
/ Oligonucleotides - administration & dosage
/ Satellite Cells, Skeletal Muscle - metabolism
/ Science
