Drug intelligence / Profile preview

endothelin-1 plasmid nanoparticle

Development stage
Preclinical
Lead developer
Northwestern University
Modality
Gene Therapies, Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

Endothelin-1 plasmid nanoparticle is an experimental gene therapy construct designed for the targeted delivery of plasmid DNA encoding the Endothelin-1 (ET-1) protein to endothelial cells. Developed by researchers at Northwestern University, the system utilizes a nanoparticle delivery vehicle to selectively express ET-1 in the pulmonary vasculature. In preclinical models of pulmonary arterial hypertension (PAH), this agent has been used to investigate the GCN2/ATF4/HIF-2a/Endothelin-1 signaling pathway. Specifically, it was shown to reverse the attenuated pulmonary hypertension phenotype in GCN2-deficient mice, confirming that endothelial ET-1 expression is a critical downstream mediator of GCN2-induced vascular remodeling.

Other names
ET-1 plasmid nanoparticleET1 plasmid nanoparticleET 1 plasmid nanoparticleEndothelial cell-targeted nanoparticle delivery of plasmid DNA expressing Endothelin-1
02

Targets

ET-1 (Endothelin-1)

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