Unlocking the potential of mRNA for the future treatment of rare diseases
Drug Discovery World
FEBRUARY 28, 2023
Now, given the extraordinary success of the Covid-19 vaccines, the mRNA platform may bring renewed hope as researchers explore its potential to advance medicines for rare diseases. Interestingly, at least 80% of all rare diseases originate from monogenic mutations, meaning that they arise from mutations in a single gene 4.
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