公开(公告)号：US20200347369A1

公开(公告)日：2020-11-05

申请号：US16845009

申请日：2020-04-09

Applicant: California Institute of Technology

Inventor： Yuki Oka ,  Allan-Hermann Pool

IPC: C12N9/22 ,  C12N15/86 ,  C12N15/11 ,  A61K48/00 ,  A61K9/00 ,  A61K35/12 ,  A61P25/28

Abstract: Disclosed herein include methods and compositions for incorporating an effector gene into the genome of a cell. The method can comprise introducing into a cell a donor nucleic acid comprising a recognition site, a splice acceptor site, a self-cleaving peptide sequence, an effector gene, and an optional transcript stabilization element. The donor nucleic acid can be incorporated into the intron of a target gene differentially expressed in a unique cell type and/or in a cell during a unique cell state via non-homologous end joining (NHEJ)-dependent DNA repair. There are also provided, in some embodiments, methods and compositions for treating a disease or disorder in a subject.