| Amyotrophic Lateral Sclerosis (ALS) | SOD1 and FUS | Human ALS patient fibroblast | Corrects the mutation A272C in SOD1 and G1566A in FUS. | Not clear if treatment after disease onset would be effective.
Reduces the expression of both wild type and mutant gene. | (51) |
| tardbp and fus | Zebrafish | Correction of missense mutations in these ALS-associated genes. | N/A* | (55) |
| Alzheimer’s Disease (AD) | Mutation in Amyloid Precursor Protein (APP) | Human | Disrupts expression of mutant APP. | Shortening the gRNA could lead to decreased on-target efficacy | (66; 75) |
| PSEN2 | Basal forebrain cholinergic neurons | Correction of the N141I mutation resulted in normalization of observed Aβ42/40 increase. | N/A* | (73) |
| PSEN2 | Human | Abolishes the electrophysiological deficit and restores the number of spikes and spike height. | N/A* | (73) |
| PSEN1 | Human | (c.236 T > C) mutation correction. | N/A* | (71) |
| PSEN1 | Human | (c.449C > T) mutation correction of the PSEN1 gene. | N/A* | (72) |
| APOE4 | Human | Converts APOE4 to APOE2 or E3.
Effective in neutralizing the risk of AD. | N/A* | (68) |
| Parkinson’s Disease (PD) | LRRK2 | Human | Corrects the p.G2019S mutation in LRRK2 and neurite complexity.
Retained pluripotency of hiPSCs after gene editing. | N/A* | (102) |
| SNCA | Human cell line | Corrects mutation in SNCA gene. | N/A* | (103) |
| Colorectal Cancer (CRC) | PAR3L | Human CaCO-2 Cells | KO results in reduced proliferation and induction of apoptosis of CRC cell line. | Study was limited to CRC cell lines, no primary cells used. | (38) |
| TP53 | Human colon adenocarcinoma-derived cell lines | Correction of mutations of TP53 at exon 3 and exon 10 may alter the malignant potential of these cells. | Not tested on all of the genomic mutations and clinical varieties of TP53. | (131) |
| APC | Human and mouse organoids | Colonoscopy-guided mucosal injection used to deliver CRISPR-engineered organoids.
Facilitates studying adenoma-carcinoma-metastasis progression. | Colonoscopy and specific surgical equipment are required. | (139) |
| KRAS | Human cell lines | sgRNA library targeting protein-coding genes in KRAS-mutant CRC cell lines used to identify genes associated with reduced tumor growth. | N/A* | (36) |
| Prostate Cancer | PD-1 | Phase I clinical trial | PD-1 knockdown of T cells in castration-resistant prostate cancer. | Confirmation of successful knockdown and a significant change in disease phenotype cannot yet be made, as the clinical trial is ongoing. | (152) |
| GPRC6A | Human cell line | Reduces primary tumor growth. | N/A* | (148) |
| Androgen receptor (AR) gene | Human Cell line | Restrains growth of androgen-dependent prostate cancer and potential therapeutic strategy for prostate cancer treatment. | Limited to androgen-dependent prostate cancer not androgen-independent prostate cancer. | (146) |
| Transcription factor NANOG andpseudogene NANOGP8 | Human cell line | Attenuates malignant potential and migration capability. | Knockout of both NANOG1 and NANOGP8 genes is lethal. | (150) |
| Breast Cancer | HER2 | Human cell line | Inhibits cell growth and tumorgenicity. | Effects downstream MAPK/ERK and PI3K/AKT signaling cascades, in non-cancer cells. | (163) |
| Pten | Mouse model | Pten silencing by lentiviral delivery results in development of invasive lobular breast cancer. | Lentiviral delivery causes immune response. | (161) |
| CDK8/19 | Human cell line | Suppress estrogen-induced gene expression in breast cancer. | N/A* | (169) |
| Ubr5 | Mice | Impairs tumor growth and metastasis. | N/A* | (170) |
| MIEN1 | Human epithelial breast cancer | Deletions of MIEN1 gene lead to the abrogation of breast cancer. | N/A* | (171) |
| Ovarian Cancer | DNMT1 | Human ovarian cancer cell line (SKOV-3) and mice | Inhibition of tumor growth | N/A* | (175; 176) |
| MTH1 | A subcutaneous xenograft tumor model of SKOV3 cells in BALB/c nude mice | Apoptosis and genetic damage of cancerous cells resulting in tumor growth inhibition. | N/A* | (177) |
| miR-21 | Human ovarian cancer cell lines (SKOV-3 & OVCAR3) | Inhibition of the epithelial-to-mesenchymal transition (EMT) in ovarian cancer cells. | N/A* | (178) |
| PARP-1 | SKOV-3 cell line and a SKOV-3 xenograft BALB/C mice model | Increased cancer cell death | N/A* | (179) |
| Rheumatoid arthritis | FOXP3-associated genes | Human Regulatory T-cell (Treg) | Augmentation of the suppressive ability of Tregs via increased Treg stability.
Insertion of chimeric antigen receptor (CAR) gene increased potency in cancer therapy. | N/A* | (181) |
| Lung infection | MUC18 | Human primary airway epithelial cells (AECs) | Reduced IL-8 (proinflammatory chemokine) responses. | Mixed population of edited cells and phenotypic changes unrelated to the gene knockout. | (12) |