Himeda Charis L, Jones Takako I, Jones Peter L
The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
Trends Pharmacol Sci. 2016 Apr;37(4):249-251. doi: 10.1016/j.tips.2016.02.001. Epub 2016 Feb 22.
Versatility of CRISPR/Cas9-based platforms makes them promising tools for the correction of diverse genetic/epigenetic disorders. Here we contrast the use of these genome editing tools in two myopathies with very different molecular origins: Duchenne muscular dystrophy, a monogenetic disease, and facioscapulohumeral muscular dystrophy, an epigenetic disorder with unique therapeutic challenges.
基于CRISPR/Cas9的平台的多功能性使其成为纠正各种遗传/表观遗传疾病的有前景的工具。在这里，我们对比了这些基因组编辑工具在两种分子起源截然不同的肌病中的应用：杜氏肌营养不良症，一种单基因疾病；以及面肩肱型肌营养不良症，一种具有独特治疗挑战的表观遗传疾病。
