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Article

Self-amplifying RNAs generated with the modified nucleotides 5-methylcytidine and 5-methyluridine mediate strong expression and immunogenicity in vivo

1 Apr 2024

Abstract excerpt

Abstract When utilized in therapeutic applications, synthetic self-amplifying RNA (saRNA) can lead to higher and more sustained expression than standard messenger RNA (mRNA). This feature is particularly important for gene replacement therapy applications where prolonged expression could reduce the dose and frequency of treatments. The inclusion of modified nucleotides in synthetic non-amplifying mRNA has been...

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PMID: 41256007
DOI: 10.1093/narmme/ugae004

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