Posted by Friends of FSH Research on Jan 6, 2025
Report by Dr. Block
See also Collaborative Effort to Evaluate a Novel Treatment Paradigm for FSHD Using EB-ON Technology
Our collaborative research project aims to use mRNA encoding our proprietary EB-ON technology, to activate proteins that suppress DUX4 activity. This approach has the potential to provide long-term DUX4 silencing without the need for permanently introducing foreign genetic material into patients. During the initial phase of the grant, we focused on optimizing mRNA and guide RNA delivery to muscle cells from FSHD patients. Despite some challenges, we successfully delivered the mRNA and activated our first set of target genes. The next phase will expand the scope of gene testing and investigate DUX4 repression within these cells.
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