For the first time, a team in Spain demonstrated that lentivirus-mediated GT can successfully restore the transcriptional program of hematopoietic stem cells and progenitor cells (HSPCs) in patients with FA, bringing them close to expressing healthy HSPCs.[19] Siegner et al[20] found that editing mutations in the FANCA gene using an adenine base editor can lead to the re-expression of FANCA proteins and reactivation of the FA pathway in edited lymphoblast-like cell lines with significant value-added advantages over unedited cells. Here, FANCA is linked to Friedreich ataxia.