| 发明名称 | Inducible cell-based model for the study of Friedreich's Ataxia | ||
| 摘要 | Isolated transduced cells exhibiting FRDA characteristics in an inducible fashion are disclosed. Isolated transduced cells comprise an expression vector having a nucleic acid sequence encoding an shRNA for frataxin protein knockdown and a heterologous expression control sequence. Additionally, methods of screening for a candidate therapeutic agent for treating Friedreich's Ataxia using isolated transduced cells are disclosed. Further, a recombinant nucleic acid construct for frataxin knockdown is disclosed that comprises a nucleic acid encoding an shRNA operably linked to a heterologous expression control sequence and expressing an shRNA molecule in a dose-responsive fashion. | ||
| 申请公布号 | US9316634(B2) | 申请公布日期 | 2016.04.19 |
| 申请号 | US201414276348 | 申请日期 | 2014.05.13 |
| 申请人 | Indiana University Research and Technology Corporation | 发明人 | Payne Ronald Mark;Babbey Clifford M. |
| 分类号 | G01N33/50;C12N15/113 | 主分类号 | G01N33/50 |
| 代理机构 | Stinson Leonard Street LLP | 代理人 | Stinson Leonard Street LLP |
| 主权项 | 1. A recombinant nucleic acid construct for frataxin knockdown comprising a nucleic acid encoding an shRNA, wherein the shRNA comprises a sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2 and SEQ ID NO: 3, wherein the shRNA is operably linked to a heterologous expression control sequence. | ||
| 地址 | Indianapolis IN US | ||