Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice |
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Authors: | Kawakami Emi Kinouchi Nao Adachi Taro Ohsawa Yutaka Ishimaru Naozumi Ohuchi Hideyo Sunada Yoshihide Hayashi Yoshio Tanaka Eiji Noji Sumihare |
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Institution: | Department of Orthodontics and Dentofacial Orthopedics, Institute of Health Bioscience, The University of Tokushima Graduate School, Japan. |
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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. |
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Keywords: | atelocollagen muscle muscular dystrophy myostatin RNA interference |
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