Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice.
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.
Department of Orthodontics and Dentofacial Orthopedics, Institute of Health Bioscience, The University of Tokushima Graduate School, Japan.
Emi
E
Kawakami
Nao
N
Kinouchi
Taro
T
Adachi
Yutaka
Y
Ohsawa
Naozumi
N
Ishimaru
Hideyo
H
Ohuchi
Yoshihide
Y
Sunada
Yoshio
Y
Hayashi
Eiji
E
Tanaka
Sumihare
S
Noji
D000818:Animals; D051244:Caveolin 3 / Q000172:deficiency* / Q000235:genetics; D003094:Collagen / Q000235:genetics / Q000378:metabolism*; D005260:Female; D008297:Male; D051379:Mice; D009133:Muscular Atrophy / Q000628:therapy*; D055435:Myostatin / Q000235:genetics*; D034622:RNA Interference; D034741:RNA, Small Interfering
D051244:Caveolin 3; D055435:Myostatin; D034741:RNA, Small Interfering; C059821:atelocollagen; D003094:Collagen