Methods and means for treating genetic disorders associated with instability of DNA repeats

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专利权人:

バイオマリン;ベー．フェー．;テクノロジーズ;バイオマリン テクノロジーズ ベー.フェー.

发明人:

デ キンペ ヨセフス ヨハネス,プラテンバーグ ヘラルド ヨハンヌ,ワンシンク デリック ヘルト

申请号：

JP2015050040

公开号：

JP5934408B2

申请日:

2015.03.12

申请国别(地区):

JP

年份:

2016

代理人:

摘要：

The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.

来源网站：

中国工程科技知识中心

来源网址：

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