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

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]