Design of RNA-Binding Proteins: Manipulate Alternative Splicing in Human Cells with Artificial Splicing Factors.
Design of RNA-Binding Proteins: Manipulate Alternative Splicing in Human Cells with Artificial Splicing Factors.
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DOI：
10.1007/978-1-4939-3591-8_18
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发表时间：
2016
影响因子：
--
通讯作者：
Yang Wang;Zefeng Wang
中科院分区：
文献类型：
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作者：
Yang Wang;Zefeng Wang
大多数人类基因经历选择性剪接以产生具有不同功能的多种异构体。选择性剪接的失调与多种人类疾病密切相关，因此调节疾病相关剪接事件的新方法将提供巨大的治疗潜力。在这里，我们报告的协议，构建新的人工剪接因子，可以被设计为专门调节靶基因的选择性剪接。通过遵循本方案中概述的方法，可以设计和产生在调节不同类型的选择性剪接中具有不同活性的人工剪接因子。人工剪接因子可用于改变培养细胞中小基因或内源基因的剪接，为研究选择性剪接的调控和选择性剪接产物的功能提供了新的策略。
The majority of human genes undergo alternative splicing to produce multiple isoforms with distinct functions. The dysregulations of alternative splicing have been found to be closely associated with various human diseases; thus new approaches to modulate disease-associated splicing events will provide great therapeutic potentials. Here we report protocols for constructing novel artificial splicing factors that can be designed to specifically modulate alternative splicing of target genes. By following the method outlined in this protocol, it is possible to design and generate artificial splicing factors with diverse activities in regulating different types of alternative splicing. The artificial splicing factors can be used to change splicing of either minigenes or endogenous genes in cultured human cells, providing a new strategy to study the regulation of alternative splicing and function of alternatively spliced products.