Funding outcome

Muscular dystrophy is a progressive genetic muscle disorder that mainly affects children, causing muscle weakness and wasting. Although the causative genes are continue to be discovered, there is no cure for the disease. One promising treatment is an antisense oligonucleotide (ASO), a synthetic molecule that can control specific RNA. ASOs have the potential to directly target the causative genes of muscular dystrophy and are considered a next-generation treatment. However, delivering ASOs effectively to the muscle cells throughout the body is challenging. This can lead to insufficient treatment effects or require high doses of ASOs, which could cause harmful side effects. To solve this problem, our research team has been exploring novel, innovative compounds that can improve the delivery of ASOs to muscle cells. In this study, we focus on two promising drug delivery molecules that we have identified in previous experiments. We plan to test the effectiveness and safety of these molecules using animal models of muscular dystrophy. Specifically, we will test a novel peptide molecule called DG9, by adding it to ASOs and using it in a dog model of Duchenne muscular dystrophy (DMD). In addition, we will test lipid nanoparticle-conjugated ASOs in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). The goal of this research is to improve the delivery of ASOs, making them safer and more effective for the treatment of muscular dystrophy. This research could pave the way for the development of new treatments for muscular dystrophy and help build a platform for future clinical applications.