MicroRNA Therapeutic Advantage (why MUSC chose RNA over gene therapy)

The MUSC team evaluated multiple approaches (gene therapy, micro-RNA therapeutics) and chose the RNA-based approach because it "offered several advantages." These likely include: - Reversibility: RNA effects wear off when the drug clears, making it safer if there are unexpected side effects - Manufacturing: RNA therapeutics are generally easier and cheaper to manufacture than viral vectors - Dosing flexibility: RNA doses can be adjusted more easily than gene therapy (which is typically one-time) - Regulatory familiarity: RNA therapeutics have a longer regulatory track record than novel AAV gene therapies Trade-off: RNA therapeutics don't provide a permanent cure — patients would need ongoing treatment. Gene therapy offers the potential of a single "curative" dose, but with higher risk and regulatory complexity.