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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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。 |
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