Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass

N. Kinouchi, Y. Ohsawa, N. Ishimaru, H. Ohuchi, Y. Sunada, Y. Hayashi, Y. Tanimoto, K. Moriyama, S. Noji

Research output: Contribution to journalArticle

55 Citations (Scopus)

Abstract

RNA interference (RNAi) offers a novel therapeutic strategy based on the highly specific and efficient silencing of a target gene. Since it relies on small interfering RNAs (siRNAs), a major issue is the delivery of therapeutically active siRNAs into the target tissue/target cells in vivo. For safety reasons, strategies based on vector delivery may be of only limited clinical use. The more desirable approach is to directly apply active siRNAs in vivo. Here, we report the effectiveness of in vivo siRNA delivery into skeletal muscles of normal or diseased mice through nanoparticle formation of chemically unmodified siRNAs with atelocollagen (ATCOL). ATCOL-mediated local application of siRNA targeting myostatin, a negative regulator of skeletal muscle growth, in mouse skeletal muscles or intravenously, caused a marked increase in the muscle mass within a few weeks after application. These results imply that ATCOL-mediated application of siRNAs is a powerful tool for future therapeutic use for diseases including muscular atrophy.

Original languageEnglish
Pages (from-to)1126-1130
Number of pages5
JournalGene Therapy
Volume15
Issue number15
DOIs
Publication statusPublished - Aug 1 2008
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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    Kinouchi, N., Ohsawa, Y., Ishimaru, N., Ohuchi, H., Sunada, Y., Hayashi, Y., Tanimoto, Y., Moriyama, K., & Noji, S. (2008). Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass. Gene Therapy, 15(15), 1126-1130. https://doi.org/10.1038/gt.2008.24