Pharmacology & ToxicologyNucleic Acid TherapeuticsPeer-reviewed
Improved efficacy and tolerability of antisense oligonucleotide with Guanidine- bridged nucleic acid
Takaaki Kawanobe, Ajaya Ram Shrestha, Hideaki Tomita
This study evaluates GuNA-modified ASOs, showing improved KD activity, long-term efficacy, and reduced toxicity in a mouse model.
DOI 10.1177/21593337261502560