RNA is the molecular "messenger" that carries instructions from DNA to the protein-making machinery of the cell. RNA therapeutics work by modifying this messaging process. For MCHS, the MUSC team pursued micro-RNA therapeutics — using small RNA molecules to either block harmful RNA messages or enhance productive ones.
How it's different from gene therapy: Gene therapy delivers a new copy of the gene (DNA) into cells. RNA therapy modifies the existing gene's message. It's like adjusting the volume on a radio rather than installing a new radio. It can be more targeted and potentially reversible, but it doesn't "fix" the underlying genetic problem the way gene therapy does.