Hangyu Wu, Aniket Wahane, Feryal Alhamadani, Kristy Zhang, Rajvi Parikh, SooWan Lee, Evan M McCabe, Theodore P Rasmussen, Raman Bahal, Xiao-Bo Zhong, José E Manautou
The field of antisense oligonucleotide (ASO)-based therapies have been making strides in precision medicine due to their potent therapeutic application. Early successes in treating some genetic diseases are now attributed to an emerging class of antisense drugs. After two decades, the US Food and Drug Administration (FDA) has approved a considerable number of ASO drugs, primarily to treat rare diseases with optimal therapeutic outcomes. However, safety is one of the biggest challenges to the therapeutic utility of ASO drugs...
December 2022: Current Opinion in Toxicology