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 2011
Externally publishedYes

Fingerprint

Caveolin 3
Myostatin
Muscular Atrophy
Small Interfering RNA
Limb-Girdle Muscular Dystrophies
RNA Interference
Gene Expression
Muscles
Therapeutics
atelocollagen

Keywords

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

ASJC Scopus subject areas

  • Developmental Biology
  • Cell Biology

Cite this

Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice. / Kawakami, Emi; Kinouchi, Nao; Adachi, Taro; Ohsawa, Yutaka; Ishimaru, Naozumi; Ohuchi, Hideyo; Sunada, Yoshihide; Hayashi, Yoshio; Tanaka, Eiji; Noji, Sumihare.

In: Development Growth and Differentiation, Vol. 53, No. 1, 01.2011, p. 48-54.

Research output: Contribution to journalArticle

Kawakami, Emi ; Kinouchi, Nao ; Adachi, Taro ; Ohsawa, Yutaka ; Ishimaru, Naozumi ; Ohuchi, Hideyo ; Sunada, Yoshihide ; Hayashi, Yoshio ; Tanaka, Eiji ; Noji, Sumihare. / Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice. In: Development Growth and Differentiation. 2011 ; Vol. 53, No. 1. pp. 48-54.
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