CORDIS Project
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This project focuses on developing a novel RNA therapeutic approach using circular RNA for long-term expression of the dystrophin protein in Duchenne muscular dystrophy. By utilizing lipid nanoparticles for targeted delivery, it aims to restore dystrophin function and provide a potential treatment for this genetic diso…
RNA therapeutics is an emerging field explored in various types of diseases such as genetic disorders, cancer, inflammation and viral infections.
Currently, most of the research focuses on the delivery of mRNA molecules that will transiently express a desired protein that can replace a defective protein or manipulate gene expression in the cells.
My lab was the first to show systemic, cell-specific delivery of mRNA molecules in animals.
Our approach and our novel amino lipids were translated to…
TEL AVIV UNIVERSITY
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