ERC Advanced Grant · 2021
Circular RNA Therapeutics for Duchenne Muscular Dystrophy
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 several clinical trials in the field of infectious and monogenic diseases. In protein replacement therapy, the main hurdle of using mRNA is the relative short half life of the mRNA. To address this,…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.