OLIGONUKLEOTID ZA LIJEČENJE PACIJENATA S MUSKULARNOM DISTROFIJOM

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专利权人:

BioMarin Technologies B.V.

发明人:

Van Deutekom, Judith Christina Theodora

申请号：

HRP20192007

公开号：

HRP20192007T1

申请日:

2019.11.06

申请国别(地区):

HR

年份:

2020

代理人:

摘要：

To provide pharmaceutical compositions comprising oligonucleotides which can preferably induce skipping of two or more exons of an mRNA precursor.SOLUTION: An oligonucleotide can bind to a first exon region derived from a dystrophin mRNA precursor and a second exon region in the same mRNA precursor, the region of the second exon has at least 50% identity to the region of the first exon, and the oligonucleotide is suitable for skipping the first exon and the second exon of the mRNA precursor, as well as preferably the entire stretch of exons in between the first and the second exons.SELECTED DRAWING: None

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/