发明名称 |
Methods and means for treating DNA repeat instability associated genetic disorders |
摘要 |
The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary onl y 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. |
申请公布号 |
AU2007282224(B2) |
申请公布日期 |
2013.08.29 |
申请号 |
AU20070282224 |
申请日期 |
2007.08.10 |
申请人 |
PROSENSA TECHNOLOGIES B.V. |
发明人 |
PLATENBURG, GERARD JOHANNES;WANSINK, DERICK GERT;DE KIMPE, JOSEPHUS JOHANNES |
分类号 |
C12N15/113;A61K31/7088;A61K41/00;A61P43/00 |
主分类号 |
C12N15/113 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|