Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

L Zangi, KO Lui, A Von Gise, Q Ma, W Ebina… - Nature …, 2013 - nature.com
L Zangi, KO Lui, A Von Gise, Q Ma, W Ebina, LM Ptaszek, D Später, H Xu, M Tabebordbar
Nature biotechnology, 2013nature.com
In a cell-free approach to regenerative therapeutics, transient application of paracrine factors
in vivo could be used to alter the behavior and fate of progenitor cells to achieve sustained
clinical benefits. Here we show that intramyocardial injection of synthetic modified RNA
(modRNA) encoding human vascular endothelial growth factor-A (VEGF-A) results in the
expansion and directed differentiation of endogenous heart progenitors in a mouse
myocardial infarction model. VEGF-A modRNA markedly improved heart function and …
Abstract
In a cell-free approach to regenerative therapeutics, transient application of paracrine factors in vivo could be used to alter the behavior and fate of progenitor cells to achieve sustained clinical benefits. Here we show that intramyocardial injection of synthetic modified RNA (modRNA) encoding human vascular endothelial growth factor-A (VEGF-A) results in the expansion and directed differentiation of endogenous heart progenitors in a mouse myocardial infarction model. VEGF-A modRNA markedly improved heart function and enhanced long-term survival of recipients. This improvement was in part due to mobilization of epicardial progenitor cells and redirection of their differentiation toward cardiovascular cell types. Direct in vivo comparison with DNA vectors and temporal control with VEGF inhibitors revealed the greatly increased efficacy of pulse-like delivery of VEGF-A. Our results suggest that modRNA is a versatile approach for expressing paracrine factors as cell fate switches to control progenitor cell fate and thereby enhance long-term organ repair.
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