Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
AQP4 antisense RNA 1 (AQP4-AS1)
- Target
- AQP4-AS1
- Molecular classification
- Long non-coding RNA (lncRNA), Other
01
Overview
AQP4 antisense RNA 1 (AQP4-AS1) is a long non-coding RNA transcribed from the antisense strand of the AQP4 gene on chromosome 18[4]. It does not encode a protein but functions as a regulator by modulating the expression of the AQP4 water channel, particularly in retinal Müller glial cells[1][5]. AQP4-AS1 expression is elevated under diabetic conditions and in diabetic retinopathy, where it promotes neurodegeneration and vascular dysfunction by downregulating AQP4 expression. Silencing of AQP4-AS1 in experimental models can alleviate retinal neurovascular impairment, indicating its potential as a therapeutic target in diabetic retinopathy[1][5]. In addition, certain SNPs in AQP4-AS1 are associated with breast cancer risk, suggesting its broader role in disease modulation[3].
- Other names
- FLJ30507C18orf16CHST9-AS1CHST9 antisense RNA 1 (non-protein coding)AQP4-AS1
02
Mechanism of action
Antisense inhibition (modifies the expression of AQP4 mRNA at the transcriptional/post-transcriptional level); RNA interference (e.g., via shRNA/siRNA targeting AQP4-AS1 for functional studies)
03
Biological functions
Regulation of gene expression (specifically AQP4 mRNA/protein)Regulation of Müller glial cell functionModulation of neurovascular unit integrityRegulation of retinal cell apoptosis and gliosis
04
Disease associations
Diabetic retinopathyBreast cancerOther neurovascular and retinal disorders (inferred from role in glial and endothelial regulation)
05
Safety considerations
Off-target effects and specificity when using RNA-targeting therapeutics (inferred from lncRNA biology, not explicitly discussed in search results)Potential impact on water homeostasis and neuronal function via indirect regulation of AQP4 expression (the key CNS water channel)[1][5]
06
Biomarkers
AQP4-AS1 expression levels (as a potential biomarker for retinal neurodegeneration and diabetic retinopathy severity[1][5])SNP rs527616 in AQP4-AS1 (associated with breast cancer risk[3])
Beyond the preview
Go deeper on AQP4 antisense RNA 1 (AQP4-AS1).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Clinical trials
Full profile accessFollow the clinical studies evaluating therapies directed at this target.
- Trial design
- Status
- Readouts
Competitive landscape
Full profile accessCompare approaches across drug candidates, modalities, and indications.
- Programs
- Modalities
- Indications
Literature & evidence
Full profile accessInvestigate the research and source evidence behind target biology and development.
- Publications
- Sources
- Analysis
Patents
Full profile accessExplore patent activity around therapies and technologies addressing this target.
- Patents
- Assignees
- Technologies
Research & analysis
Full profile accessConnect target biology, drug development, and emerging evidence in your research.
- Biology
- Development news
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AQP4 antisense RNA 1 (AQP4-AS1).