Article

Anticodon Edited Transfer RNAs (ACE-tRNAs) Encoded as Therapeutic Nonviral Minimal DNA Vectors

2025-09-06

Abstract excerpt

Nonsense mutations, resulting from a premature termination codon (PTC), make up ~11% of all genetic lesions causing disease, affecting millions of people worldwide. Nonsense suppressor anticodon-edited tRNAs (ACE-tRNAs) have emerged as a therapeutic modality for the rescue of PTCs. Delivery of ACE-tRNAs in vivo has been achieved by adeno-associated viral vector and RNA-lipid nanoparticle, however due to drawbacks...

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Literature Corpus work: 89f26cff-0b74-534a-bce9-1e6fbf7379d3
DOI: 10.1101/2025.09.06.674645

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