RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.
RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.
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DOI：
10.2174/156652312802083585
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发表时间：
2012-08
影响因子：
3.6
通讯作者：
Harper SQ
中科院分区：
文献类型：
--
作者：
Liu J;Harper SQ
肢体束带肌营养不良症（LGMD）是指一组由25种常见临床特征相关的遗传性疾病，包括支撑骨盆和肩带的肌肉萎缩。心脏也可能受累。与其他肌肉萎缩症一样，LGMDs目前是无法治愈的，但针对隐性形式的前瞻性基因替代疗法在临床前和临床研究中显示出希望。相比之下，很少有人关注开发针对显性LGMD的基因治疗方法，这可能会受益于疾病基因沉默。尽管迄今为止对开发显性lgmd的基因疗法缺乏关注，但该领域并非从头开始，因为对隐性lgmd的转化研究提供了一个框架，可以应用于治疗显性形式的疾病。在这篇文章中，我们讨论了用基因沉默方法治疗显性遗传形式的LGMD的前景。
Limb Girdle Muscular Dystrophy (LGMD) refers to a group of 25 genetic diseases linked by common clinical features, including wasting of muscles supporting the pelvic and shoulder girdles. Cardiac involvement may also occur. Like other muscular dystrophies, LGMDs are currently incurable, but prospective gene replacement therapies targeting recessive forms have shown promise in pre-clinical and clinical studies. In contrast, little attention has been paid to developing gene therapy approaches for dominant forms of LGMD, which would likely benefit from disease gene silencing. Despite the lack of focus to date on developing gene therapies for dominant LGMDs, the field is not starting at square one, since translational studies on recessive LGMDs provided a framework that can be applied to treating dominant forms of the disease. In this manuscript, we discuss the prospects of treating dominantly inherited forms of LGMD with gene silencing approaches.