FOXF1 minicircle DNA nanoparticles

FOXF1 minicircle DNA nanoparticles (npFOXF1) is an experimental non-viral gene therapy designed to restore the expression of the FOXF1 transcription factor in pulmonary endothelial cells. The therapy utilizes biodegradable nanoparticles, specifically a PEI600-MA5/PEG-OA/Cho formulation, to deliver minicircle plasmid DNA encoding the human FOXF1 gene under the control of an EF1α promoter. FOXF1 is a critical transcription factor for lung development and vascular integrity; its deficiency or mutation is the primary cause of Alveolar Capillary Dysplasia with Misaligned Pulmonary Veins (ACDMPV), a rare and fatal congenital lung disorder. Preclinical research conducted at Cincinnati Children's Hospital and the University of Arizona has demonstrated that npFOXF1 can rescue endothelial injury and improve survival in models of ACDMPV and transplant-associated lung complications, such as idiopathic pneumonia syndrome.

Other names

minicircle FOXF1 nanoparticlesFOXF1 minicircle DNA-loaded nanoparticlesFOXF-1 minicircle DNA-loaded nanoparticlesFOXF 1 minicircle DNA-loaded nanoparticles