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用於治療肌肉萎縮症患者的寡核苷酸
专利权人:
PROSENSA TECHNOLOGIES B.V.
发明人:
JUDITH CHRISTINA THEODORA VAN DEUTEKOM
申请号:
HK15110181.1
公开号:
HK1209456A
申请日:
2015.10.16
申请国别(地区):
HK
年份:
2016
代理人:
摘要:
The invention relates to an oligonucleotide and to a pharmaceutical composition comprising said oligonucleotide. This oligonucleotide is able to bind to a region of a first exon from a dystrophin pre-mRNA and to a region of a second exon within the same pre-mRNA, wherein said region of said second exon has at least 50% identity with said region of said first exon, wherein said oligonucleotide is suitable for the skipping of said first and second exons of said pre-mRNA, and preferably the entire stretch of exons in between.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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