TY - JOUR AU - Pérez Luz, Sara AU - Gimenez-Cassina, Alfredo AU - Fernández-Frías, Iván AU - Wade-Martins, Richard AU - Díaz-Nido, Javier PY - 2015 DO - 10.1016/j.ygeno.2015.05.006 UR - https://hdl.handle.net/20.500.14352/102046 T2 - Genomics AB - Friedreich's ataxia (FRDA) is the most common form of hereditary ataxia caused by recessive mutations in the FXN gene. Recent results have indicated the presence of different frataxin isoforms due to alternative gene expression mechanisms. Our... LA - eng M2 - 76 PB - Elsevier KW - Frataxin KW - Friedreich’s ataxia KW - Gene therapy KW - Bacterial artificial chromosomes KW - HSV-1 amplicons TI - Delivery of the 135 kb human frataxin genomic DNA locus gives rise to different frataxin isoforms TY - journal article VL - 106 ER -