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CGI-58 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- 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
01
Overview
CGI-58 antisense oligonucleotide is an experimental RNA-targeted agent designed to inhibit the expression of Comparative Gene Identification-58 (CGI-58), also known as Abhydrolase Domain Containing 5 (ABHD5). CGI-58 is a critical co-activator of adipose triglyceride lipase (ATGL) and is essential for the mobilization of neutral lipids from intracellular lipid droplets. In research conducted by Yale University and Ionis Pharmaceuticals, this antisense oligonucleotide (ASO) is used to study the role of lipid droplet dynamics in liver disease. Preclinical studies in mouse models of metabolic dysfunction-associated steatohepatitis (MASH) have demonstrated that knockdown of CGI-58 leads to increased lipid droplet size, elevated cholesterol content, and the promotion of liver inflammation and fibrosis, thereby serving as a model to understand the progression of MASH and identify potential therapeutic counter-targets like CIDEB or GPAM.
- Other names
- CGI-58 ASOCGI58 ASOCGI 58 ASOABHD5 antisense oligonucleotideABHD-5 antisense oligonucleotideABHD 5 antisense oligonucleotideComparative Gene Identification-58 antisense oligonucleotide
02
Targets
ABHD5 (Abhydrolase domain-containing protein 5)
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