CN Patent
CN112779252B — 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸
Assigned to Capital Institute of Pediatrics · Expires 2023-05-26 · 3y expired
What this patent protects
本发明提出靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸,所述的反义寡核苷酸能够结合至SMN2基因启动子区的MeCP2富集的甲基化区域来促进脊髓性肌萎缩症患者细胞中SMN2基因的整体转录水平和SMN蛋白的表达水平,可以作为SMN靶向性治疗策略中的新治疗靶点。
USPTO Abstract
本发明提出靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸,所述的反义寡核苷酸能够结合至SMN2基因启动子区的MeCP2富集的甲基化区域来促进脊髓性肌萎缩症患者细胞中SMN2基因的整体转录水平和SMN蛋白的表达水平,可以作为SMN靶向性治疗策略中的新治疗靶点。
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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