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] |