[EN] COMPOUNDS FOR THE REDUCTION OF THE DELETERIOUS ACTIVITY OF EXTENDED NUCLEOTIDE REPEAT CONTAINING GENES<br/>[FR] COMPOSÉS POUR LA RÉDUCTION DE L'ACTIVITÉ DÉLÉTÈRE DE GÈNES CONTENANT UNE RÉPÉTITION DE NUCLÉOTIDES ÉTENDUE
申请人:NUREDIS INC
公开号:WO2020131573A1
公开(公告)日:2020-06-25
Aspects of the present disclosure include methods of reducing the deleterious impact of a target gene in a cell, such as the deleterious activity of a mutant extended nucleotide repeat (NR) containing target gene in a cell by contacting the cell with an effective amount of a tetrahydrocarbazole compound. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended NR containing target gene may be reduced, e.g., by reducing (and in some instances differentially, including selectively, reducing) the production or activity of toxic expression products (e.g., RNA or protein) encoded by the target gene. Kits and compositions for practicing the subject methods are also provided.
本公开内容的方面包括减少目标基因在细胞中的有害影响的方法,例如通过将细胞与有效量的四氢咔唑化合物接触,减少含有突变延伸核苷酸重复序列(NR)的目标基因在细胞中的有害活性。突变延伸NR包含的目标基因的有害活性(例如,由此编码的产物的毒性和/或功能障碍)可以减少,例如,通过减少(在某些情况下包括选择性地减少)由目标基因编码的有毒表达产物(例如,RNA或蛋白质)的生产或活性。还提供了用于实施所述方法的试剂盒和组合物。