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Abstract
Incorporation of N<sup>1</sup>-methyladenosine1-methyladenosine** in suppressor transfer RNAs improved readthrough of premature termination codons.
- Suppressor transfer RNAs (sup-tRNAs) can promote readthrough of disease-causing premature termination codons.
- Chemical modification of sup-tRNAs enhances their activity by improving aminoacylation and functional persistence.
- Optimized pulmonary lipid nanoparticles (LNPs) effectively deliver chemically modified sup-tRNAs to lung tissues.
- Restoration of cystic fibrosis transmembrane conductance regulator (CFTR) expression and function was observed in bronchial epithelial cells and mouse models.
- This approach suggests a potential therapeutic platform for treating nonsense mutations.
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mRNA Technology