Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
The ATP-binding cassette transporter A1 (ABCA1) mRNA 3'-untranslated region (3'-UTR) is a key regulatory element that governs the post-transcriptional expression of the ABCA1 protein (UniProt: P94187). ABCA1 plays a fundamental role in cellular lipid homeostasis by facilitating the efflux of cholesterol and phospholipids to apolipoprotein A-I (apoA-I), which is the rate-limiting step in the biogenesis of high-density lipoprotein (HDL) particles. The 3'-UTR of ABCA1 is notably long and contains conserved binding sites for several microRNAs, most significantly miR-33a/b, which act to suppress ABCA1 translation and promote mRNA decay (Rayner et al., Science, 2010; PMID: 20466885). Therapeutic strategies targeting this region involve using antisense oligonucleotides or microRNA inhibitors to block these inhibitory interactions, thereby increasing ABCA1 protein levels (Rotllan et al., J Lipid Res, 2016; PMID: 26839344). Upregulation of ABCA1 via its 3'-UTR is a major area of research for treating atherosclerosis, as it promotes reverse cholesterol transport and reduces the formation of foam cells in arterial walls. Additionally, this target is being investigated for its potential role in neurodegenerative diseases like Alzheimer's, where ABCA1-mediated lipid transport influences amyloid-beta clearance (Koldamova et al., J Biol Chem, 2014; PMID: 24610481).
Inhibition of microRNA binding (specifically miR-33) to the 3'-UTR to prevent translational repression and mRNA degradation, leading to increased ABCA1 protein expression and enhanced cholesterol efflux (Rayner et al., Science, 2010; PMID: 20466885).
3 more in the full profile.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Explore the programs pursuing this target and their development progress.
Follow the clinical studies evaluating therapies directed at this target.
Compare approaches across drug candidates, modalities, and indications.
Investigate the research and source evidence behind target biology and development.
Explore patent activity around therapies and technologies addressing this target.
Connect target biology, drug development, and emerging evidence in your research.
See how Gosset can support your research on ATP-binding cassette transporter A1 mRNA 3'-untranslated region (ABCA1 mRNA 3'-UTR) (ABCA1 mRNA 3'-UTR).