ＤＮＡリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段,申请号JP20090523740-传众专利搜索

ＤＮＡリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段

申请公布号：JP6047270(B2)

申请号：JP20090523740

申请日期：2007.08.10

申请公布日期：2016.12.21

申请人：

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

分类号：C12N15/09;C12N15/113;C12Q1/68

主分类号：C12N15/09

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.