Unlocking the potential of mRNA for the future treatment of rare diseases
Drug Discovery World
FEBRUARY 28, 2023
mRNA technology helped us to vaccinate the world against SARS-CoV-2. Approximately 7,000 rare and ultra-rare diseases have been described, and yet, 90% have no approved therapeutic options 1. Therefore, the risk for off-target insertional mutagenesis is greatly reduced.
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