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Pkd1 ASO
- Development stage
- Preclinical
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous, Intracerebroventricular, Intraperitoneal, Intravenous, Intra-arterial, Intracardiac, Intrahypothalamic, Intraportal, Systemic
01
Overview
Pkd1 ASO is a 16-base pair antisense oligonucleotide designed to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD). It functions by sterically binding to the microRNA-17 binding motif (P1MBM) located within the Pkd1 3’ untranslated region (3’UTR). This binding prevents miR-17 mediated translational repression of Pkd1 mRNA, leading to its stabilization and an increase in polycystin 1 (PC1) protein levels. In preclinical studies, Pkd1 ASO has been shown to stabilize Pkd1 mRNA, increase PC1 protein, slow cyst growth, and improve metabolism in both Pkd1 RC/- mouse models and human ADPKD kidney cyst-derived cells.
02
Targets
Polycystin 1 (PKD1) mRNA 5' untranslated region (UTR) upstream open reading frame (uORF)
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