Target intelligence / Profile preview

PAX6 antisense RNA 1 (PAX6-AS1)

Target: PAX6-AS1
Molecular classification: Long non-coding RNA (lncRNA), Antisense RNA, Other (not a protein, receptor, or enzyme)

Overview

PAX6 antisense RNA 1 (PAX6-AS1) is a long non-coding RNA transcribed from the Pax6 locus in an antisense direction, overlapping intron 1 of Pax6. It is especially expressed in pancreatic beta cells and islets, and its expression is elevated by high glucose and in type 2 diabetes. Functionally, PAX6-AS1 regulates beta cell identity and function, influencing insulin and PDX1 gene expression, and modulates glucose-stimulated insulin secretion. Mechanistically, it binds to EIF3D and histones H3/H4, suggesting roles in mRNA translation and chromatin regulation. Silencing PAX6-AS1 in human beta cells enhances insulin secretion, while overexpression impairs it, making it a promising candidate as a therapeutic target and biomarker for Type 2 diabetes. No direct drugs currently target PAX6-AS1, but antisense RNA and CRISPR strategies have been used in experimental research[1].

Other names: Pax6os1PAX6-AS1lncRNA-PAX6-AS1

Biological functions

Regulation of beta cell gene expressionModulation of insulin transcription/secretionInteraction with translation machinery and histone modificationEpigenetic regulation

Disease associations

Type 2 diabetes (associated with altered expression in diabetic islets)Metabolic dysfunction (potential role in beta cell dysfunction)Other (no strong links to cancer or classic inflammation/neurological roles)

Safety considerations

Off-target effects possible if therapeutically silencing/overexpressing in non-beta cells (preclinical evidence only)Species-specific and sex-specific effects observed; further validation needed for human therapeutics

Biomarkers

PAX6-AS1 expression (potential biomarker for beta cell dysfunction and Type 2 diabetes)

Beyond the preview

Go deeper on PAX6 antisense RNA 1 (PAX6-AS1).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Explore the programs pursuing this target and their development progress.

Drug candidates
Developers
Development stage

Clinical trials

Follow the clinical studies evaluating therapies directed at this target.

Trial design
Status
Readouts

Competitive landscape

Compare approaches across drug candidates, modalities, and indications.

Programs
Modalities
Indications

Literature & evidence

Investigate the research and source evidence behind target biology and development.

Publications
Sources
Analysis

Patents

Explore patent activity around therapies and technologies addressing this target.

Patents
Assignees
Technologies

Research & analysis

Connect 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 PAX6 antisense RNA 1 (PAX6-AS1).

Explore the full profile