Table 3:
Correction of pathogenic mutations in vivo using CRISPR/ Cas9
| Disease | Species | Type of mutation | Reference | |
|---|---|---|---|---|
| 1. | Duchenne muscular dystrophy (DMD) | Mouse | Dmd gene correction | 68, 69, 76, 77 |
| 2. | Tay-Sachs or Sandhoff disease | Mouse | HEXA and HEXB gene correction | 70 |
| 3. | Sandhoff disease | iPS cells/ Organoids | HEXB gene correction | 71 |
| 4. | Tay-Sachs and Sandhoff Diseases | Mouse | HEXA and HEXB gene correction | 72 |
| 5. | Tyrosinemia type I and mucopolysaccharidosis type I | Mouse | Allelic exchange in Fah and Idua genes | 73 |
| 6. | Mucopolysaccharidosis type II (MPS II) | Mouse | Ids gene correction | 74 |
| 7. | Duchenne muscular dystrophy (DMD) | Mouse | Permanent exon skipping in Dmd gene | 75 |
| 8. | Interstitial lung disease | Mouse | Inactivation of Sftpc gene | 78 |