<i>In vivo</i> efficacy and safety of systemically administered serinol nucleic acid-modified antisense oligonucleotides in mouse kidney.

ABSTRACT: Nucleic acid medicine encompassing antisense oligonucleotides (ASOs) has garnered interest as a potential avenue for next-generation therapeutics. However, their therapeutic application has been constrained by challenges such as instability, off-target effects, delivery issues, and immunogenic responses. Furthermore, their practical utility in treating kidney diseases remains unrealized. Recently, we developed a serinol nucleic acid-modified ASO (SNA-ASO) that exhibits significant nuclease resistance. In this study, we evaluated the in vivo efficacy of SNA-ASOs in mouse kidney. We subcutaneously administered various types of phosphorothioate-modified gapmer ASOs with SNA or 2'-O-methoxyethyl (2'-MOE) modifications (MOE-ASO) targeting sodium glucose cotransporter 2 (SGLT2) in

SUBMITTER: Tsuboi T 

PROVIDER: S-EPMC11754010 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature