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

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 () and Daniela Palacios ()
Additional contact information
Francesco Millozzi: Università Cattolica del Sacro Cuore
Paula Milán-Rois: IMDEA Nanociencia
Arghya Sett: Inserm U1212
Giovanni Delli Carpini: Università Cattolica del Sacro Cuore
Marco De Bardi: Fondazione Santa Lucia IRCCS
Miguel Gisbert-Garzarán: IMDEA Nanociencia
Martina Sandonà: Università Cattolica del Sacro Cuore
Ciro Rodríguez-Díaz: IMDEA Nanociencia
Mario Martínez-Mingo: IMDEA Nanociencia
Irene Pardo: IMDEA Nanociencia
Federica Esposito: Sapienza University of Rome
Maria Teresa Viscomi: Università Cattolica del Sacro Cuore
Marina Bouché: Sapienza University of Rome
Ornella Parolini: Università Cattolica del Sacro Cuore
Valentina Saccone: Università Cattolica del Sacro Cuore
Jean-Jacques Toulmé: Inserm U1212
Álvaro Somoza: IMDEA Nanociencia
Daniela Palacios: Università Cattolica del Sacro Cuore

Nature Communications, 2025, vol. 16, issue 1, 1-19

Abstract: Abstract 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.

Date: 2025
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DOI: 10.1038/s41467-024-55223-9

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