...:基因置换就是用正常的基因原位替换病变细胞内的致病基因,使细胞内的DNA完全恢复正常状态。这种治疗方法最为理想,但目前由于技术原因尚难达到。 基因修复(gene correction):基因修复是指将致病基因的突变碱基序列纠正,而正常部分予以保留。
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The ultimate goal of gene therapy is correction of the genetic disorders by introducing the genetic materials into targeted cells, and gene expression should be permanent and at an appropriate level.
基因治疗的目的是将遗传物质导入细胞并使之得到适宜水平的表达,以纠正机体的遗传缺陷,恢复细胞的正常功能或杀死癌细胞及致病微生物。
Targeted gene expression to the epidermis has been achieved, but was neither a prerequisite for correction nor did it improve longevity of expression.
皮肤靶向基因的表达已完成测序,但这并不是我们就能对它进行修饰的先决条件。
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