Morpholino-driven gene editing: A new horizon for disease treatment and prevention.
Morpholino-driven gene editing: A new horizon for disease treatment and prevention.
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DOI：
10.1002/cpt.276
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发表时间：
2016-01
影响因子：
6.7
通讯作者：
Zingman LV
中科院分区：
文献类型：
--
作者：
Subbotina E;Koganti SR;Hodgson-Zingman DM;Zingman LV
遗传和分子生物学工具的发展使特定基因与其功能相联系，加速了我们对潜在健康和疾病的分子途径的理解。由此带来的知识进步将基因靶向推向了有希望的治疗策略的前沿。在这里，我们讨论了独特的强大和适应性的方法，吗啉驱动的正常和突变基因表达的修改作为一条通往健康的途径。
The development of genetic and molecular biology tools permitting the connection of specific genes to their functions has accelerated our understanding of molecular pathways underlying health and disease. The resulting gains in knowledge have propelled gene targeting to the forefront of promising therapeutic strategies. Here we discuss the uniquely powerful and adaptable approach of morpholino-driven modification of normal and mutant gene expression as a pathway to health.