Mass Spectrometry Imaging Reveals the Distribution of a GABRG2 Targeting Antisense Oligonucleotide and Its Functional Effect in Rat Brain.

ABSTRACT: In recent years, the development of antisense oligonucleotides (ASOs) has gained wide interest as therapeutic agents for their potential in treating neurodegenerative diseases. ASOs are chemically modified oligonucleotides that are designed to bind complementary regions of RNA or DNA and, thereby, modulate the expression of the corresponding protein. Here, we present a multiomics approach to investigate the spatial distribution and biological effect of an ASO designed to target the mRNA that translates for γ-aminobutyric acid A receptor γ2 subunit (GABRG2), which is abundantly expressed within the brain. In this study, a rat model was used to develop a multiomics mass spectrometry (imaging) approach to map ASO distribution in brain and kidney, followed by in-depth analysis of the lipidome,

SUBMITTER: van der Vloet L 

PROVIDER: S-EPMC12532189 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature