发明名称 |
CRISPR-based methods and products for increasing frataxin levels and uses thereof |
摘要 |
Methods and products (e.g., gRNAs, recombinant fusion proteins, frataxin targeting systems, compositions and kits) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom. |
申请公布号 |
AU2015234204(A1) |
申请公布日期 |
2016.10.06 |
申请号 |
AU20150234204 |
申请日期 |
2015.03.20 |
申请人 |
Universite Laval |
发明人 |
Tremblay, Jacques P.;Chapdelaine, Pierre;Rousseau, Joel |
分类号 |
C12N15/113;A61K47/48;A61P9/00;A61P25/28;C07H21/02;C07K14/22;C07K14/315;C07K14/47;C07K19/00;C12N15/11;C12N15/12;C12N15/62;C12N15/63;C12N15/86 |
主分类号 |
C12N15/113 |
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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