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INDUCIBLE CELL-BASED MODEL FOR THE STUDY OF FRIEDREICH'S ATAXIA
INDUCIBLE CELL-BASED MODEL FOR THE STUDY OF FRIEDREICH'S ATAXIA
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机译:基于单元格的弗里德里希·阿塔克斯病研究模型
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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.
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