ヘモグロビン(HBF/HBG)に対する天然アンチセンス転写物の抑制によるHBF/HBG関連疾患の治療

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专利权人:

クルナ・インコーポレーテッド

发明人:

ジョゼフ・カラード,オルガ・コルコヴァ・シャーマン,カルロス・コイト

申请号：

JP2016083203

公开号：

JP6407912B2

申请日:

2016.03.31

申请国别(地区):

JP

年份:

2018

代理人:

摘要：

PROBLEM TO BE SOLVED: To provide therapeutic means of diseases associated with hemoglobin (HBF/HBG) by the inhibition of natural antisense transcript against HBF/HBG.SOLUTION: The present invention relates to e.g., a method of regulating function and/or expression of globin polynucleotides in patients cells or tissues in vivo, the method comprising contacting the cells or tissues with an antisense compound 5-30 nucleobases in length specific to a nucleic acid molecule antisense strand of a globin polynucleotide comprising at least one oligonucleotide having 20% complementarity to at least five consecutive nucleic acids of an antisense or sense nucleic acid molecule of the globin polynucleotide.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/