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miRDOE iPS-MSCs

Development stage
Preclinical
Lead developer
Kaohsiung Chang Gung Memorial Hospital
Modality
Gene Therapies, iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intra-arterial
01

Overview

miRDOE iPS-MSCs is an experimental cell-based gene therapy consisting of human induced pluripotent stem cell-derived mesenchymal stem cells (iPS-MSCs) genetically engineered to doubly overexpress two microRNAs: miR-19a-3p and miR-20a-5p. This therapeutic approach, referred to as miRDOE (microRNA double overexpression), is designed to enhance the cytoprotective and regenerative properties of MSCs. Preclinical research in rat models has demonstrated that these engineered cells are superior to non-modified iPS-MSCs in preserving renal function and architecture in the context of chronic kidney disease (CKD) complicated by acute ischemia-reperfusion (IR) injury. The therapy works by downregulating multiple pathological pathways, including oxidative stress (NOX-1, NOX-2, NOX-4), inflammatory signaling (TLR2, TLR4, NF-κB), and apoptotic markers (caspase-3, Bax).

Other names
miR-19a-3p and miR-20a-5p doubly overexpressing iPS-MSCsmiR19a-3p and miR-20a-5p doubly overexpressing iPS-MSCsmiR 19a-3p and miR-20a-5p doubly overexpressing iPS-MSCsmiRDOE of iPS-MSCsmiRDOE-iPS-MSCs
02

Targets

NF-κBBAX (Apoptosis regulator BAX)CASP3 (Caspase-3)NOX4 (NADPH oxidase 4)

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