Oligonucleotides Containing Acyclic Nucleoside Analogues with Carbamate Internucleoside Linkages
摘要:
Synthesis of 2'-deoxy-2',3'-secothymidine t and its dimer t*t, where the two 2'-deoxy-2',3'-secothymidine t units are connected via a carbamate, *=3'-NH-CO-O-5', internucleoside linkage has been achieved. These building blocks were protected in the 5'-position, converted into their phosphoramidites, or attached onto CPG, and then used for ''chimeric oligonucleotide'' synthesis.
[EN] COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF<br/>[FR] COMPOSITIONS POUR L'ÉDITION DE TRANSCRITS MECP2 ET PROCÉDÉS ASSOCIÉS
申请人:[en]WAVE LIFE SCIENCES LTD.
公开号:WO2023049477A2
公开(公告)日:2023-03-30
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.