Compositions for editing MECP2 transcripts and methods thereof

In addition to this, the disclosure provides oligonucleotides, compositions and methods thereof that can achieve specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions, and methods are useful for treating, preventing, or ameliorating MECP2-related disord...

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Hauptverfasser: YANG HAILIN, MARAPPAN, SUBRAMANIAN, KIVATAKARN, ONAN, YU HUI, VARGHESE, CHANDRA, LU GENLIANG, KANDASAMY, PARCHAMUTHU, GODFREY JACK DAVID, HARDING IAN CHANDLER, LAMARTINA ANTHONY, KUMARASAMY, JAYAKANTHAN, SHIMIZU MAMORU, STANDLEY STEFANIE MICHELLE, TURNER JESSE, BLY DAVID JOHN, HERSH, ANDREW, GUZIO, SHIVALILA, CHAKDU, SHAKETI, IWAMOTO NAOKI, MONIAN, PRASHANT, BUTLER DAVID CHARLES DONNELL, DESAI, GIGA, APPONI LUCIANO ENRIQUE, AKE CHRISTOPHER MICHAEL
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:In addition to this, the disclosure provides oligonucleotides, compositions and methods thereof that can achieve specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions, and methods are useful for treating, preventing, or ameliorating MECP2-related disorders, diseases, and syndromes that can benefit from adenosine modification. 除此之外,本披露提供了可以实现靶RNA分子中的靶腺苷的特异性编辑的寡核苷酸、其组合物和方法。这样的寡核苷酸、组合物以及方法可用于治疗、预防或改善可以从腺苷修饰中受益的MECP2相关障碍、疾病和综合征。