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

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.

• Authors: Emi Kawakami, Nao Kinouchi, Taro Adachi, Yutaka Ohsawa, Naozumi Ishimaru, Hideyo Ohuchi, Yoshihide Sunada, Yoshio Hayashi, Eiji Tanaka, Sumihare Noji
• Journal: Development, growth & differentiation (Dev Growth Differ) Vol. 53 Issue 1 Pg. 48-54 (Jan 2011) ISSN: 1440-169X [Electronic] Japan
• PMID: 21261610 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2011 The Authors. Journal compilation © 2011 Japanese Society of Developmental Biologists.

Chemical References

• Caveolin 3
• Myostatin
• RNA, Small Interfering
• atelocollagen
• Collagen

Topics

• Animals
• Caveolin 3 (deficiency, genetics)
• Collagen (genetics, metabolism)
• Female
• Male
• Mice
• Muscular Atrophy (therapy)
• Myostatin (genetics)
• RNA Interference
• RNA, Small Interfering