COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF

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 di...

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Hauptverfasser: LAMATTINA, Anthony, MARAPPAN, Subramanian, BUTLER, David Charles Donnell, SHIVALILA, Chikdu Shakti, MONIAN, Prashant, IWAMOTO, Naoki, KUMARASAMY, Jayakanthan, YU, Hui, BOULAY, David John, SHIMIZU, Mamoru, TURNER, Jesse, STANDLEY, Stephany Michelle, CHIVATAKARN, Onanong, GODFREY, Jack David, HARDING, Ian Chandler, ACKER, Christopher Michael, VARGEESE, Chandra, LU, Genliang, HOSS, Andrew Guzior, KANDASAMY, Pachamuthu, APPONI, Luciano Henrique, YANG, Hailin, DESAI, Jigar
Format: Patent
Sprache:eng
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Zusammenfassung: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.