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GalNAc-conjugated antisense oligonucleotide targeting Dhcr24
- Development stage
- Preclinical
- Lead developer
- University of the Basque Country
- 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, Subcutaneous
01
Overview
GalNAc-conjugated antisense oligonucleotide targeting Dhcr24 is a preclinical-stage antisense oligonucleotide (ASO) designed to selectively inhibit the expression of 24-dehydrocholesterol reductase (DHCR24) in hepatocytes [1299]. DHCR24 is a key terminal enzyme in cholesterol biosynthesis that catalyzes the conversion of desmosterol to cholesterol. By conjugating the ASO to N-acetylgalactosamine (GalNAc), the therapeutic is specifically targeted to hepatocytes via the asialoglycoprotein receptor (ASGPR), allowing for localized gene silencing. In preclinical models of colorectal liver metastasis (CRLM), hepatocyte-specific inhibition of Dhcr24 leads to the accumulation of desmosterol and promotes CRLM progression, suggesting that modulating desmosterol metabolism in hepatocytes plays a critical role in regulating tumor cell proliferation [1299].
- Other names
- GalNAc-DHCR24 ASOGalNAc-DHCR-24 ASOGalNAc-DHCR 24 ASOGalNAc-conjugated DHCR24 ASOGalNAc-conjugated antisense oligonucleotide targeting DHCR24
02
Targets
DHCR24 (3β-hydroxysterol Δ24-reductase)
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