| abstract |
Provided herein are chimeric minigenes, wherein alternative splicing of the minigene determines whether the encoded gene is expressed. In particular, the minigene is alternatively spliced in response to the splicing modifier drug, and thus the encoded gene is expressed only in the presence of the splicing modifier drug. The encoded gene may encode a repressor RNA, a CRISPR-Cas9 protein, a transactivator or a therapeutic protein. |