Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia models.

33 , 34 In the brain, transplantation of HSPCs transduced with highdose, but not lowdose, LV-FXN virus increased aconitase activity in transplanted versus untransplanted FRDA mice, although the difference did not reach statistical significance ( Figure 4 E).

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