Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for usher syndrome

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专利权人:

发明人:

Michelle L Hastings

申请号：

US14705579

公开号：

US09556434B2

申请日:

2015.05.06

申请国别(地区):

US

年份:

2017

代理人:

摘要：

The present invention provides a method for treating Ushers syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.

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中国工程科技知识中心

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