Jeong Uk Choi

Delivery architectures for oligonucleotide therapeutics in genetic and rare diseases: GalNAc-based hepatocyte targeting and extrahepatic translation.

Many genetic and rare diseases are molecularly defined but remain difficult to treat because their biology involves intracellular targets, toxic gain-of-function mechanisms, splicing defects, repeat expansions, or chemically undruggable pathways. Although RNA medicines can be designed directly from genetic information,...