Sweet move: a modified sugar enhances antisense oligonucleotide safety and efficacy

Researchers found that adding a newly developed modified sugar, BNAP-AEO, to gapmer antisense oligonucleotides (ASOs) increased their affinity for target RNAs, thus significantly enhancing their gene-silencing effects in vitro and in vivo. The BNAP-AEO modification also decreased gapmer ASO toxicity to the central nervous system (CNS), suggesting that it could improve the clinical application of ASO treatment of CNS disease.