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splice-switching antisense oligonucleotide targeting UMOD exon 10
- Development stage
- Preclinical
- Lead developer
- Indiana University
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
Splice-switching antisense oligonucleotide targeting UMOD exon 10 (also known as Umod SSO or SSO (-13)) is an antisense oligonucleotide designed to modulate the alternative splicing of uromodulin (UMOD) pre-mRNA. By targeting the 5' splice site of UMOD exon 10, the oligonucleotide induces exon skipping, leading to the expression of an intracellular, non-GPI-anchored uromodulin isoform (AS-UMOD). This isoform localizes to the mitochondria, where it interacts with solute carrier family 25 (SLC25) transporters to enhance mitochondrial metabolism, increase NAD+ and ATP levels, and protect renal thick ascending limb (TAL) cells from hypoxic and ischemic stress. Developed by researchers at Indiana University, this therapeutic candidate is being investigated for the treatment of acute kidney injury (AKI).
- Other names
- Umod SSOsplice-switching oligonucleotide targeting UMOD exon 10
02
Targets
UMOD (Uromodulin)
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