In vivo gene therapies succeed or fail based on their ability to achieve the right level of gene expression in the right cells. Delivery vehicles such as adeno associated viruses (AAVs) determine where the genetic cargo arrives, but it is the promoter that dictates what happens next, whether a therapeutic gene is active and to what extent. As gene therapy enters a new phase of clinical maturity, promoter engineering remains undervalued, even though it is central to addressing the two defining challenges of targeting and dosing.
This article explores the pivotal role of promoters in determining therapeutic protein expression and why their design is critical for the success of gene therapies.
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