TTR and Leber congenital amaurosis 10: The FDA has approved the clinical application of CRISPR-based gene therapies for the treatment of sickle cell disease (SCD) [194], β-thalassemia (TDT) [195], transthyretin (TTR) amyloidosis [196], Leber congenital amaurosis type 10 (LCA10) [197], human immunodeficiency virus (HIV) [198], and Duchenne muscular dystrophy (DMD) [199].