PMES-miR-200a

PMES-miR-200a is a plasmid-based gene therapy candidate designed to over-express microRNA-200a (miR-200a) for the treatment of craniosynostosis, including syndromic forms like Saethre-Chotzen syndrome. Developed by researchers at the University of Iowa, the therapy utilizes the Plasmid-based microRNA Expression System (PMES) delivered via PEGylated-peptide nanoparticles. In genetic models of Twist1 mutation, where miR-200a levels are typically insufficient to prevent premature suture fusion, PMES-miR-200a restores miR-200a expression. This over-expression helps maintain the population of Gli1 and Six2 positive suture stem cells and regulates osteogenic pathways to prevent the premature fusion of cranial sutures, thereby allowing for normal brain and neural development.