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

Author

Listed:
  • Francesco Millozzi

    (Università Cattolica del Sacro Cuore
    Sapienza University of Rome)

  • Paula Milán-Rois

    (IMDEA Nanociencia)

  • Arghya Sett

    (Inserm U1212
    Luxembourg Institute of Science and Technology (LIST))

  • 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
    Fondazione Santa Lucia IRCCS)

  • Ciro Rodríguez-Díaz

    (IMDEA Nanociencia)

  • Mario Martínez-Mingo

    (IMDEA Nanociencia)

  • Irene Pardo

    (IMDEA Nanociencia)

  • Federica Esposito

    (Sapienza University of Rome
    Fondazione Santa Lucia IRCCS)

  • Maria Teresa Viscomi

    (Università Cattolica del Sacro Cuore
    Universitario Agostino Gemelli IRCCS)

  • Marina Bouché

    (Sapienza University of Rome)

  • Ornella Parolini

    (Università Cattolica del Sacro Cuore
    Universitario Agostino Gemelli IRCCS)

  • Valentina Saccone

    (Università Cattolica del Sacro Cuore
    Universitario Agostino Gemelli IRCCS)

  • Jean-Jacques Toulmé

    (Inserm U1212
    Novaptech)

  • Álvaro Somoza

    (IMDEA Nanociencia
    Centro Nacional de Biotecnología (CNB-CSIC))

  • Daniela Palacios

    (Università Cattolica del Sacro Cuore
    National Research Council (CNR))

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.

Suggested Citation

  • 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 & F, 2025. "Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles," Nature Communications, Nature, vol. 16(1), pages 1-19, December.
    • Handle: RePEc:nat:natcom:v:16:y:2025:i:1:d:10.1038_s41467-024-55223-9
    DOI: 10.1038/s41467-024-55223-9
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