An engineered UGA suppressor tRNA gene for disease-agnostic AAV delivery
Xu M, Liu H, Wang J, Vieira A, Zhou X, Liu N, Liang J, Du A, Chen X, Xing R, Yang Y, Gonzalez-Perez M, Kumar V, Wang D. An engineered UGA suppressor tRNA gene for disease-agnostic AAV delivery. Nature Biotechnology 2026, 1-10. PMID: 41555020, DOI: 10.1038/s41587-025-02982-5.Peer-Reviewed Original Research
Recombinant adeno-associated virusRAAV vector productionAdeno-associated virusPathogenic nonsense variantIn vivo deliveryC-to-T transitionLysosomal storage disorderAAV deliveryMouse modelPotential therapeutic scopeNonsense variantNonsense mutationTherapeutic approachesTherapeutic effectGenes in vivoNormal levelsSuppressor tRNA geneUGA stop codonStorage disorderSuppressor transfer RNATranscriptional regulatory elementsCGA arginine codonTherapeutic scopeTRNA genesTransfer RNA