Research field | hiPSCs-derived cells | CRISPR/Cas9 gene editing | Outcome | Ref. | |
---|---|---|---|---|---|
Drug development | Evaluation of PEPT1-mediated intestinal absorption | Intestinal epithelial-like cells | Peptide transporter 1 (PEPT1)-knock-out iPSCs | Setting the basis for the development of peptide and peptide-mimetic drugs as possible substrates of PEPT1 | [65] |
Multiple-system atrophy (OMIM #146500) | Neurons | Correction of COQ2 mutation | Identification of Q10 as possible therapeutic target | [66] | |
Monitoring of protein dosage | FOXG1 syndrome | Interneurons | Tag FOXG1 gene with small molecule-assisted shut-off (sMASh) | Demonstration of FOXG1 dose-control | [67] |
Gene therapy | Beta-thalassemia (OMIM #613985) | Hematopoietic stem cells | Correction of HBB mutation | Corrected-hematopoietic stem cells transplantation as therapeutic strategy | |
Recessive dystrophic epidermolysis bullosa (OMIM #226600) | Keratinocytes and fibroblasts | Correction of COL7A1 mutation | Restoration of the regular collagen type VII expression | [70] | |
Duchenne muscular dystrophy (OMIM #310200) | Skeletal muscle cells | DMD exon 44 knock-in | Restoration of full protein coding-region | [71] | |
Drug screening | mtDNA depletion syndrome (OMIM #251880) | Hepatocytes | Inducing DGUOK knock-out | Identification of compound able to restore mithocondrial function | [72] |
Alzheimer’s disease (OMIM #104300) | Neurons | Correction of PSEN1 G384A mutation | Identification of a synergistic combination of bromocriptine, cromolyn and topiramate as an anti-Aβ cocktail | [73] | |
Immune response strategy | HIV infection | Macrophages | Introduction of 32bp-depletion in CCR5 gene | Generation of immune cells resistant to HIV-infection | [74] |
HIV infection | Monocytes/macrophages | Engineer hiPSCs to express a CRISPR/Cas9 system directed against the reverse-transcribed products of the viral RNA genome | Stable expression of HIV-targeted CRISPR/Cas9 in hiPSCs-derived reservoir cells | [75] | |
SARS-Cov-2 infection | Pneumocytes type II | Regulation of genes involved in viral infection | Building a cell platform to test the capacity of candidate antiviral compounds | [76] | |
Solid tumors | Natural killer | hiPSCs were edited with CRISPR/Cas9 to repress ADAM17 expression | Obtaining natural killer cells directed against tumor cells | [77] |