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Which single data gap (PK, dose-ranging efficacy, or long term safety) would most change your stance if answered convincingly

Unlike larger proteins, their small size allows them to bind to cell receptors with high specificity making them ideal for targeted therapeutic use

& Gesbeck, M

It has been established that it is capable of up- and downregulating at least 4,000 genes in the human genome, essentially resetting DNA back to a healthier state [22]
