Aug 2026
Nonviral delivery of chemically modified tRNA rescues nonsense mutations in cystic fibrosis.
This work combined site-specific chemical modification of sup-tRNAs with cargo-tailored pulmonary lipid nanoparticle (LNP) engineering to overcome barriers and restore cystic fibrosis transmembrane conductance regulator (CFTR) expression and function.
Jin-Gan Chen, Mu-Ye Zhou, Song-Tao Dong et al.
· Science · 2 citations