阅读说明：本技术 基因表达的选择性剪接调控及治疗方法 (Alternative splicing regulation of gene expression and therapeutic methods ) 是由 B·L·戴维森 A·M·莫内斯 A·A·亨德利 P·T·拉努姆 于 2019-08-07 设计创作，主要内容包括：本文提供了嵌合反式激活子小基因,其中小基因的选择性剪接决定了反式激活子是否表达。反式激活子的表达导致受设计启动子序列控制下的靶基因转录。或者,本文提供嵌合靶基因小基因,其中小基因的选择性剪接直接决定靶基因是否表达。靶基因可以编码抑制性RNA、CRISPR-Cas9蛋白或治疗性蛋白。(Provided herein are chimeric transactivator minigenes, wherein alternative splicing of the minigene determines whether the transactivator is expressed. Expression of the transactivator results in transcription of the target gene under the control of the designed promoter sequence. Alternatively, provided herein are chimeric target gene minigenes, wherein alternative splicing of the minigene directly determines whether the target gene is expressed. The target gene may encode an inhibitory RNA, a CRISPR-Cas9 protein, or a therapeutic protein.)