FBN1 and amyotrophic lateral sclerosis: Although, due to technical limitations, the overall efficiency of this knock-in application remains much lower as compared to the generation of knockout mutations, the generation of such patient-specific genetic avatars has been accomplished in TDP43/ALS (Armstrong et al., 2016), in FBN1 in various heritable connective tissue disorders (HCTD) (Yin et al., 2021), RPS14 in myelodysplastic syndrome (MDS) (Ear et al., 2016).