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<p>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.</p>

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Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年1月10日

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  • Francesco Millozzi,
  • Paula Milán-Rois,
  • Arghya Sett,
  • Giovanni Delli Carpini,
  • Marco De Bardi,
  • Miguel Gisbert-Garzarán,
  • Martina Sandonà,
  • Ciro Rodríguez-Díaz,
  • Mario Martínez-Mingo,
  • Irene Pardo,
  • Federica Esposito,
  • Maria Teresa Viscomi,
  • Marina Bouché,
  • Ornella Parolini,
  • Valentina Saccone,
  • Jean-Jacques Toulmé,
  • Álvaro Somoza,
  • Daniela Palacios

摘要

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.