For an autologous translational approach, we utilized CRISPR (Clustered Regularly interspaced short-palindromic repeat) - Cas9 (CRISPR-associated 9) gene editing technology to excise the GAA trinucleotide expansion from intron 1 of FXN in FRDA patients’ CD34+ HSPCs. Here, CD34 is linked to Friedreich ataxia.