CFTR and cystic fibrosis: Furthermore, the SMaRT method has also been employed to correct mutations in genetic diseases such as cystic fibrosis (CF) and haemophilia A. In CF, different SMaRT strategies have demonstrated to be efficient in correcting the ΔF508 mutation in CF transmembrane conductance regulator (CFTR) gene [222,223,224,225], while in a haemophilia A knockout mice model, a pre-trans-splicing molecule has been able to rectify endogenous FVIII (coagulation factor VIII) mRNA [226].