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Table 6 Overview of gene correction of genetic diseases using CRISPR/Cas9

From: CRISPR/Cas9: the Jedi against the dark empire of diseases

Genetic disease Method of CRISPR/Cas9 delivery Conclusion/outcome Reference
Tyrosinemia Tail vein hydrodynamic injection into adult mice Correction of Fah gene mutation (1 nt substitution) [83]
Hemophillia A Transfection based delivery into iPSCs Inversion based correction of the blood coagulation factor VIII (F8) gene [84]
Hemophillia B Tail vein hydrodynamic injection into Fah mice Correction of mutation in F9 gene [85]
Cataract Injection into Oocyte of mouse Correction in mutation of CRYGC gene (1 nt insertion) [77]
Sickle cell anemia Adenovirus based transduction into human IPSCs Correction in sixth codon of beta globin gene [81]
Beta Thalassemia Transfection and piggyback removal in IPSCs from patients HBB mutations corrected (1 nt substitution 4 nt insertion) [79]
Cystic fibrosis Transfection into intestinal stem cells from patients Correction of CFTR gene mutation (3 substitution) [50]