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基因编辑工具减少小鼠的阿尔茨海默病斑块前体
SPLICER 采用了一种称为外显子跳过的基因编辑方法,这种方法对于由产生错误折叠或有毒蛋白质的突变所引起的健康状况特别感兴趣,如杜氏肌营养不良症 (Duchenne’s muscular dystrophy) 或亨廷顿病 (Huntington’s disease)。
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