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Transplantation of hMSCs Genome Edited with LEF1 Improves Cardio-Protective Effects in Myocardial Infarction
Hyun-Min Cho1, Kang-Hoon Lee1, Yi-Ming Shen2
1Department of Biochemistry, BK21 PLUS Program for Creative Veterinary Science Research and Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul, South Korea.
Molecular Therapy. Nucleic Acids
|February 20, 2020
Summary
This study shows that genetically engineered stem cells expressing LEF1 improve survival and heart function after myocardial infarction (MI) in rats. This novel stem cell therapy offers a promising treatment for cardiovascular disease.
Area of Science:
- Regenerative Medicine
- Cardiovascular Research
- Gene Therapy
Background:
- Ischemic heart diseases, like myocardial infarction (MI), represent a significant global health burden.
- Stem cell-based therapy is a promising avenue for treating MI.
- Enhancing stem cell properties is crucial for effective therapeutic outcomes.
Purpose of the Study:
- To evaluate the cardio-protective effects of human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) engineered to overexpress lymphoid enhancer-binding factor 1 (LEF1).
- To assess the therapeutic potential of LEF1-overexpressing hUCB-MSCs (LEF1/hUCB-MSCs) in a rat model of MI.
Main Methods:
- LEF1 gene was integrated into hUCB-MSCs using CRISPR/Cas9-mediated gene editing at the AAVS1 safe harbor locus.
- LEF1/hUCB-MSCs were transplanted into the infarction region of rats post-MI.
- Cardio-protective effects were assessed using echocardiography, histology, and immunohistochemistry.
Main Results:
- LEF1 overexpression enhanced hUCB-MSC proliferation and survival under hypoxic conditions.
- Transplantation of LEF1/hUCB-MSCs significantly improved overall survival rates in the MI rat model.
- Echocardiogram parameters (EF, FS), infarct size, and fibrosis were markedly improved by LEF1/hUCB-MSC treatment.
Conclusions:
- LEF1/hUCB-MSCs demonstrate significant cardio-protective effects in a rat MI model.
- Enhanced stem cell survival and proliferation, along with growth factor secretion by LEF1, contribute to therapeutic benefits.
- Combining stem cell therapy with genome editing for transcription factor LEF1 presents a viable strategy for cardiovascular disease treatment.
