公开(公告)号：US20230340498A1

公开(公告)日：2023-10-26

申请号：US17798851

申请日：2021-02-12

申请人： The Children's Hospital of Philadelphia

发明人： Beverly L. DAVIDSON ,  Alejandro Mas MONTEYS ,  Amiel A. HUNDLEY ,  Paul T. RANUM

IPC分类号： C12N15/62 ,  A61K48/00 ,  C12N15/86 ,  C07K14/015

CPC分类号： C12N15/625 ,  A61K48/005 ,  C07K14/015 ,  C12N15/86 ,  C07K2319/02 ,  C12N2320/33 ,  C12N2750/14143

摘要： Provided herein are chimeric minigenes, where the alternative splicing of the minigene determines whether an encoded gene is expressed. In particular, the minigenes are alternatively spliced in response to splicing modulator dmgs, such that the encoded gene is only expressed in the present of the splicing modulator dmg. The encoded gene may encode an inhibitory RNA, a CRISPR-Cas9 protein, a transactivator, or a therapeutic protein.