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OGT ASO3
- Development stage
- Preclinical
- Lead developer
- University of Kansas Medical Center
- 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
- Intraperitoneal
01
Overview
OGT ASO3 is an antisense oligonucleotide (ASO) designed to target and knock down the expression of O-GlcNAc transferase (OGT). Developed by researchers at The University of Kansas Medical Center, OGT ASO3 is being investigated for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). In preclinical mouse models of ADPKD, administration of OGT ASO3 reduced O-GlcNAc levels in the kidney, markedly attenuated renal cyst growth, decreased kidney inflammation and fibrosis, and preserved kidney function.
- Other names
- Ogt ASO3
02
Targets
OGT (UDP-N-acetylglucosamine—peptide N-acetylglucosaminyltransferase)
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