HBB and early-onset autosomal dominant Alzheimer disease: Other diseases associated with single base substitutions, such as DMD (DMD gene)39,40, achondroplasia (FGFR3 gene)45, Alzheimer’s disease (PSEN2 gene)46, retinitis pigmentosa (Pde6b gene)47, sickle cell disease (HBB gene)48, and hemophilia B (HBB gene)37, have also been targeted with ssODN-templated gene correction.