CN Patent

CN113444722B — 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用

Assigned to Center for Excellence in Brain Science and Intelligence Technology Chinese Academy of Sciences · Expires 2024-09-24 · 2y expired

What this patent protects

本发明涉及单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用，首次利用单碱基编辑介导的剪接修复技术，针对SMN2基因的7号外显子进行编辑，通过对该外显子部分碱基的变异，成功地提高了功能性SMN2蛋白的表达。

USPTO Abstract

本发明涉及单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用，首次利用单碱基编辑介导的剪接修复技术，针对SMN2基因的7号外显子进行编辑，通过对该外显子部分碱基的变异，成功地提高了功能性SMN2蛋白的表达。

Drugs covered by this patent

Patent Metadata

Patent number
CN113444722B
Jurisdiction
CN
Classification
—
Expires
2024-09-24
Drug substance claim
No
Drug product claim
No
Assignee
Center for Excellence in Brain Science and Intelligence Technology Chinese Academy of Sciences
Source
FDA Orange Book + USPTO grounding via Google Patents

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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