METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
- 专利权人:
- 发明人:
- Sergey V. Paushkin,Nikolai A. Naryshkin,Ellen Welch
- 申请号：
- US14621051
- 公开号：
- US20150283115A1
- 申请日:
- 2015.02.12
- 申请国别(地区):
- US
- 年份:
- 2015
- 代理人:
- 摘要：
- Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Dystrophy.
- 来源网站：
- 中国工程科技知识中心
