Among the latest gene therapies, gene therapy strategies, such as ZFN, CRISPR, and CRISPR base editors, have been commonly applied for treating genetic diseases including Duchenne muscular dystrophy, Fanconi anemia, beta-thalassemia, et al., and diseases arising from LRRK2 G2019S mutation [1,16,42,43,44]. Here, LRRK2 is linked to Beta-thalassemia.