Wooree Ko

This paper demonstrates that DNA-based ACE-tRNA minivectors effectively suppress nonsense mutations in CFTR and REP1, with improved bioavailability and stability compared to plasmid DNA vectors.

This study optimizes ACE-tRNA function to suppress nonsense mutations, reducing the required amount by ~16-fold for common cystic fibrosis-causing PTCs.

The paper demonstrates that ACE-tRNAs can suppress nonsense mutations in CF by rescuing CFTR transcript abundance and channel function in multiple models.

Anticodon engineered tRNAs suppress CFTR nonsense mutations, increasing mRNA expression and channel function in human cells.