Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice
Atelocollagen-mediated systemic administration of myostatin-targeting siRNA improves muscular atrophy in caveolin-3-deficient mice
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DOI：
10.1111/j.1440-169x.2010.01221.x
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发表时间：
2011-01-01
影响因子：
2.5
通讯作者：
Noji, Sumihare
中科院分区：
文献类型：
--
作者：
Kawakami, Emi;Kinouchi, Nao;Noji, Sumihare
小干扰RNA（siRNA）介导的基因表达沉默正迅速成为分子治疗的有力工具。然而，siRNA的快速降解及其有限的活性持续时间需要有效的递送方法。去端胶原（ATCOL）介导的siRNA施用是治疗疾病（包括肌肉萎缩）的有前景的方法。在此，我们报告了ATCOL介导的肌肉生长抑制素靶向siRNA全身给药到小窝蛋白-3缺陷型肢带型肌营养不良1C（LGMD 1C）小鼠模型中诱导肌肉质量显著增加和收缩力显著恢复。这些结果提供了证据，表明ATCOL介导的SiRNA全身给药可能是治疗疾病（包括肌肉萎缩）的有力治疗工具。
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.