NEWSROOM

Researchers at the Institute of Science Tokyo have developed Chol-HDO[PMO], a cholesterol-conjugated heteroduplex oligonucleotide pairing a phosphorodiamidate morpholino oligomer with complementary RNA. Delivered into cerebrospinal fluid, it silenced or modulated target genes in mice with up to 16-fold greater potency than conventional antisense designs, and the effect was long lasting. It avoided acute neurotoxicity, weight loss and immune reactions.