| 发明名称 | Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN | ||
| 摘要 | The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Frataxin (FXN), in particular, by targeting natural antisense polynucleotides of Frataxin (FXN). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of FXN. | ||
| 申请公布号 | US9593330(B2) | 申请公布日期 | 2017.03.14 |
| 申请号 | US201214124886 | 申请日期 | 2012.06.08 |
| 申请人 | CuRNA, Inc. | 发明人 | Collard Joseph;Khorkova Sherman Olga |
| 分类号 | C07H21/02;C07H21/04;A61K31/70;C12N15/113;C12N5/00 | 主分类号 | C07H21/02 |
| 代理机构 | CuRNA, Inc. | 代理人 | CuRNA, Inc. ;Browder Monte R. |
| 主权项 | 1. A method of upregulating a function of and/or the expression of a Frataxin (FXN) polynucleotide having SEQ ID NO: 1 in a mammal comprising: contacting said system with at least one antisense oligonucleotide 10 to 30 nucleotides in length wherein said at least one oligonucleotide has at least 90% sequence identity to a reverse complement of a natural antisense of a Frataxin (FXN) polynucleotide selected from SEQ ID NO: 2; thereby upregulating a function of and/or the expression of the Frataxin (FXN) polynucleotide to treat Freidreich's ataxia in said mammal. | ||
| 地址 | Miami FL US | ||