Already, CRISPR/Cas9-based clinical trials have proven successful for sickle cell disease, beta-thalassemia, and transthyretin amyloidosis (Frangoul et al., 2021; Gillmore et al., 2021), and many efforts are focused on optimizing the conditioning regimen, such as the development of anti-CD117 antibodies that deplete HSCs in a targeted and safe manner (Kwon et al., 2019). The gene discussed is KIT; the disease is Beta-thalassemia.