Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina
Irene Vázquez-Domínguez,
Alejandro Allo Anido,
Lonneke Duijkers
et al.
Abstract:Antisense oligonucleotides (AONs) are a versatile tool for treating inherited retinal diseases. However, little is known about how different chemical modifications of AONs can affect their biodistribution, toxicity, and uptake in the retina. Here, we addressed this question by comparing splice-switching AONs with three different chemical modifications commonly used in a clinical setting (2′O-methyl-phosphorothioate (2-OMe/PS), 2′O-methoxyethyl-phosphoriate (2-MOE/PS), and phosphorodiamidite morpholino oligomer… Show more
Set email alert for when this publication receives citations?
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.