Publication number: 20250135036
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.
Type:
Application
Filed:
September 26, 2022
Publication date:
May 1, 2025
Inventors:
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