Table 1.
CRISPR-Cas9 Research and Its Application for the Treatment of Different Human Diseases
| Disease Type | Disease-Related Gene/Protein | Remarks | References |
|---|---|---|---|
| Cancer | additional sex combs like 1 (ASXL1) | CRISPR-Cas9 was used to decrease leukemia cell growth in mouse xenografts | 39 |
| myeloid cell leukemia 1 (MCL-1) | CRISPR-Cas9 was used to delete MCL-1 in human BL cells and induce apoptosis in the BL cells | 40 | |
| cyclin-dependent kinase 11 (CDK11) | CRISPR-Cas9 was used to silence CDK11 in osteosarcoma | 41 | |
| SHC SH2-binding protein 1 (SHCBP1) | SHCBP1 inhibits the proliferation of breast cancer through CRISPR-Cas9 | 42 | |
| Kelch domain containing 4 (KLHDC4) | CRISPR-Cas9 was used to knock out the KLHDC4 gene in a nasopharyngeal carcinoma cell line | 43 | |
| epidermal growth factor receptor (EGFR) | CRISPR-Cas9 was used for possible correction of acquired drug-resistant mutations in EGFR | 44 | |
| Allergy | melanoma cell adhesion molecule (MCAM/MUC18) | CRISPR-Cas9 technology was used to knock out cell surface glycoprotein MUC18 in human primary nasal airway epithelial cells | 45 |
| Janus kinase 3 (JAK3) | CRISPR-Cas9 was used to re-establish the development of normal T cells in JAK3-deficient cells | 46 | |
| Duchenne muscular dystrophy | dystrophin | CRISPR-Cas9 was used to fix DMD gene mutation for the DMD disorder | 47 |
| Cardiovascular diseases | proprotein convertase subtilisin/kexin type 9 (PCSK9) | CRISPR-Cas9 was used to correct the PCSK9 gene in an atherosclerosis mouse model | 48 |
| Huntington disease | huntingtin (HTT) gene | CRISPR-Cas9 was used to suppress the mHTT gene selectively in a mouse model | 49 |
| Alzheimer’s disease | presenilin 1 (PSEN1) and presenilin 2 (PSEN2) genes | CRISPR-Cas9 was used to correct ancestral mutations in AD related to the PSEN gene | 50,51 |
| Metabolic liver disease | Pahenu2 | CRISPR-Cas9 was used to correct the Pahenu2 gene in metabolic liver disease | 52 |
| Fanconi anemia | 17 Fanconi anemia (FA) | CRISPR-Cas9 was used to correct Fanconi anemia | 53 |
| Hereditary tyrosinemia | fumarylacetoacetase (Fah) | CRISPR-Cas9 was used to correct the Fah mutation in mouse models | 54 |
| Sickle cell anemia | β-globin gene | CRISPR-Cas9 was used to treat sickle cell disease patient blood | 55 |
| β-Thalassemia | hemoglobin subunit beta (HBB) gene | CRISPR-Cas9 was used to corrected the HBB gene mutation in human iPSCs from β-thalassemia patients | 56 |
| Cystic fibrosis | cystic fibrosis transmembrane conductance regulator (CFTR) gene | CRISPR-Cas9 was used to correct the CFTR gene in cultured stem cells of cystic fibrosis patients | 57 |
| Retinitis pigmentosa | RP1, RHO, and RPGR genes | CRISPR-Cas9 was used to interrupt the Rho (S334) mutation | 58 |
| Cataract | αA-crystallin gene | CRISPR-Cas9 was used to study the relationship of αA-crystallin mutations and human congenital cataracts | 59 |
| Human immunodeficiency virus (HIV) | long terminal repeats (LTRs) in HIV | CRISPR-Cas9 was used as a tool to mutate LTRs of HIV-1 DNA | 60 |
| Hepatitis B virus (HBV) | covalently closed circular DNAs (cccDNAs) in HBV | CRISPR-Cas9 was used to target cccDNAs of HBV | 61 |
| Human papilloma virus (HPV) | HPVE6 gene | CRISPR-Cas9 was used to target HPVE6 for cancer treatment | 62 |
| Epstein-Barr virus (EBV) | ephrin receptor tyrosine kinase A2 (EphA2) | using the EphA2 extracellular domain, a therapeutic strategy was developed | 63 |