公开(公告)号：US20230329201A1

公开(公告)日：2023-10-19

申请号：US18022509

申请日：2021-08-23

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Hailin Yang ,  Prashant Monian ,  Chikdu Shakti Shivalila ,  Subramanian Marappan ,  Chandra Vargeese ,  Pachamuthu Kandasamy ,  Genliang Lu ,  Hui Yu ,  David Charles Donnell Butler ,  Luciano Henrique Apponi ,  Mamoru Shimizu ,  Stephany Michelle Standley ,  David John Boulay ,  Jack David Godfrey ,  Naoki Iwanmoto

IPC: A01K67/027 ,  C12N15/85 ,  C12N9/78 ,  A61K49/00

CPC classification number: A01K67/0275 ,  A61K49/0008 ,  C12N9/78 ,  C12N15/8509 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/01 ,  C12N2015/8518

Abstract: Among other things, the present disclosure provides cells and non-human animals engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, the present disclosure provides cells and non-human animals engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, non-human animals are genetically modified rodents such as mice, rat, etc. In some embodiments, non-human animals are mice. In some embodiments, the present disclosure provides technologies for assessing an agent comprising administering the agent to a cell or non-human animal engineered to express an ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, such a cell or non-human animal is engineered to express a human ADAR1 polypeptide or a characteristic portion thereof. In some embodiments, an agent is a pharmaceutical agent. In some embodiments, an agent is or comprises an oligonucleotide.