公开(公告)号：US20250135036A1

公开(公告)日：2025-05-01

申请号：US18695348

申请日：2022-09-26

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： Christopher Michael Acker ,  Onanong Chivatakarn ,  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 ,  Andrew Guzior Hoss ,  Jigar Desai ,  Jack David Godfrey ,  Hailin Yang ,  Naoki Iwamoto ,  Jayakanthan Kumarasamy ,  Anthony Lamattina ,  Ian Chandler Harding ,  Jesse Turner

IPC: A61K48/00 ,  C12N9/78 ,  C12N15/113 ,  C12N15/87

Abstract: Among other things, the present disclosure provides oligonucleotides, compositions and methods thereof that can bring about specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders, diseases and syndromes that can benefit from adenosine modification.