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Transient Correction of the Phenotype in a Cardiac Mouse Model of Friedreich Ataxia Using AAV9-CAG-FXN: Toxic Effect of Supraphysiological Expression of the Frataxin Transgene

Alain Martelli,Xian Chen,Basel Assaf,Tiffany Ma,Tania Franks,Whiteley Laurence,Renea Gooch,Hongru Zhou,Vahid Farrokhi,Chuenlei Parng,Kevin Le,Ting-Wen Cheng,Ricardos Tabet,Robert Bell,Dinesh Hirenallur Shanthappa,Sheila Rao-Dayton,Patricia Lewis,Sumana Choudhury, Viktoriya G. Alekseeva,Naoko Murakami,Kimberly Zaraspe,Angela Johnson, Michelle Quiles,Allison Lewis, Joanna Cheek, Doug McCarty,Marko Pregel

MOLECULAR THERAPY（2020）

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