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 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. |
申请公布号 |
US2010184833(A1) |
申请公布日期 |
2010.07.22 |
申请号 |
US20070377160 |
申请日期 |
2007.08.10 |
申请人 |
PROSENTA TECHNOLOGIES B.V. |
发明人 |
DE KIMPE JOSEPHUS JOHANNES;PLATENBURG GERARD JOHANNES;WANSINK DERICK GERT |
分类号 |
A61K31/7052;A61K48/00;A61P43/00;C07H21/00;C12N15/00;C12N15/113;C12N15/63;C12Q1/68 |
主分类号 |
A61K31/7052 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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