RNA Therapeutics

**What it means:** 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 MHS, 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.
Search terms for this concept: existing gene's message micro-RNA therapeutics new copy of the gene