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ARHGEF6 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- AstraZeneca
- 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
01
Overview
ARHGEF6 antisense oligonucleotide is an antisense oligonucleotide (ASO) being developed by AstraZeneca for the treatment of chronic kidney disease (CKD), including diabetic kidney disease (DKD) / diabetic nephropathy. It is designed to target and reduce the expression of Rho guanine nucleotide exchange factor 6 (ARHGEF6), a RAC1-activating guanine nucleotide exchange factor. In diabetic kidney disease, dysregulated Rho GTPase signaling and hyperactive RAC1/CDC42 disrupt cytoskeletal control, leading to podocyte foot process effacement, endothelial injury, and albuminuria. By knocking down ARHGEF6, the ASO aims to restore podocyte cytoskeletal integrity, reduce urinary albumin-to-creatinine ratio (UACR), and improve glomerular pathology.
- Other names
- ARHGEF6 ASOARHGEF-6 ASOARHGEF 6 ASOArhgef6 antisense oligonucleotideArhgef-6 antisense oligonucleotideArhgef 6 antisense oligonucleotide
02
Targets
CDC42 (Cell division control protein 42 homolog)RAC1 (Rac family small GTPase 1)GEF/GAP (Ras GEF / GAP family)
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