Unlocking the potential of mRNA for the future treatment of rare diseases
Drug Discovery World
FEBRUARY 28, 2023
Interestingly, at least 80% of all rare diseases originate from monogenic mutations, meaning that they arise from mutations in a single gene 4. Therefore, the risk for off-target insertional mutagenesis is greatly reduced. In addition, it may be easier to target the therapeutic molecule to the correct cell or tissue.
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