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Table 2 Summary of the applications of CRISPR/Cas9 on hiPSCs for the identifications of therapeutic strategies

From: Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

  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 [68, 69]
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]
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Human Genomics

ISSN: 1479-7364

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