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Table 5 The tests of modified MSCs using CRISPR-Cas9 technology

From: Challenges and advances in clinical applications of mesenchymal stromal cells

Source of MSCs

Gene

Outcome

References

Human umbilical cord-derived MSCs

MCP-1/CCL2

CCL2-overexpressing hUC-MSCs showed better functional recovery relative to naïve hUC-MSCs, promoting subsequent endogenous brain repair

[134]

Human pancreatic ductal tissue MSCs

PTEN gene

PTEN mRNA synthesized in vitro is capable of being applied to a MSC-mediated anticancer strategy for the treatment of glioblastoma patients

[135]

Mouse bone marrow MSCs

SV40T into a safe harboring site at Rosa26 locus

CRISPR/Cas9 HDR-mediated immortalization of BMSCs can be more effectively reversed than that of retrovirus-mediated random integrations

[136]

Human bone marrow MSCs

Promotor of ectodysplasin (EDA)

After transfection with sgRNA-guided dCas9-E, the BM-MSCs acquired significantly higher transcription and expression of EDA by doxycycline (Dox) induction

[137]

Mouse bone marrow-derived MSCs

IL-10

Transplantation of CRISPR system engineered IL10-overexpressing bone marrow-derived MSCs for the treatment of myocardial infarction in diabetic mice

[138]

Rat bone marrow MSCs

Smad7

Smad7-MSCs is effective in treating liver fibrosis in the CCl4-induced liver cirrhosis model via inhibition of TGF-β1 signaling pathway

[139]

Human mesenchymal stem cells

First intron of the PPP1R12C gene

exogenous gene hFIX was effectively expressed following site‑specific targeting into the AAVS1 locus in MSCs; MSCs may be used as potential cell carriers for gene therapy of hemophilia B

[140]

Immortalized human bone marrow MSC cell line (ATCC PCS-500–041)

PUMILIO2 (PUM2)

Depletion of PUM2 blocks MSC adipogenesis and enhances osteogenesis. PUM2 works as a negative regulator on the 3′ UTRs of JAK2 and RUNX2 via direct binding. CRISPR/CAS9-mediated gene silencing of Pum2 inhibited lipid accumulation and excessive bone formation

[141]

Human bone marrow-MSCs

Platelet-derived growth factor B (PDGF-B)

PDGFB-MSCs increased anti-apoptotic signaling and exhibited enhanced survival and expansion after transplantation, resulting in an enlarged humanized niche cell pool that provide a better humanized microenvironment to facilitate superior engraftment and proliferation of human hematopoietic cells

[142]