Santiago Tijaro‐Bulla

Nonviral delivery of chemically modified tRNA rescues nonsense mutations in cystic fibrosis

Suppressor transfer RNAs (sup-tRNAs) can rescue disease-causing nonsense mutations by promoting readthrough of premature termination codons (PTCs). Their clinical translation is limited by suboptimal activity and inefficient in vivo delivery. In this work, we combined site-specif...