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OLIGONUCLÉOTIDE POUR LE TRAITEMENT DE PATIENTS ATTEINTS DE DYSTROPHIE MUSCULAIRE
专利权人:
BioMarin Technologies B.V.
发明人:
申请号:
EP13739847.5
公开号:
EP2870246B1
申请日:
2013.07.03
申请国别(地区):
EP
年份:
2019
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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