Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice

Emi Kawakami, Nao Kinouchi, Taro Adachi, Yutaka Ohsawa, Naozumi Ishimaru, Hideyo Ohuchi, Yoshihide Sunada, Yoshio Hayashi, Eiji Tanaka, Sumihare Noji

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

Small interfering RNA (siRNA)-mediated silencing of gene expression is rapidly becoming a powerful tool for molecular therapy. However, the rapid degradation of siRNAs and their limited duration of activity require efficient delivery methods. Atelocollagen (ATCOL)-mediated administration of siRNAs is a promising approach to disease treatment, including muscular atrophy. Herein, we report that ATCOL-mediated systemic administration of a myostatin-targeting siRNA into a caveolin-3-deficient mouse model of limb-girdle muscular dystrophy 1C (LGMD1C) induced a marked increase in muscle mass and a significant recovery of contractile force. These results provide evidence that ATCOL-mediated systemic administration of siRNAs may be a powerful therapeutic tool for disease treatment, including muscular atrophy.

Original languageEnglish
Pages (from-to)48-54
Number of pages7
JournalDevelopment Growth and Differentiation
Volume53
Issue number1
DOIs
Publication statusPublished - Jan 1 2011
Externally publishedYes

Keywords

  • Atelocollagen
  • Muscle
  • Muscular dystrophy
  • Myostatin
  • RNA interference

ASJC Scopus subject areas

  • Developmental Biology
  • Cell Biology

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