Target intelligence / Profile preview

SYNGAP1 antisense RNA 1 (SYNGAP1-AS1)

Target
SYNGAP1-AS1
Molecular classification
Long non-coding RNA (lncRNA), Natural antisense transcript (NAT), Non-coding RNA
01

Overview

SYNGAP1 antisense RNA 1 (SYNGAP1-AS1) is a long non-coding RNA transcribed in the antisense direction to the SYNGAP1 gene. It does not code for a protein but acts as a regulatory NAT (natural antisense transcript) that can negatively regulate SYNGAP1 mRNA and protein levels by epigenetic or transcriptional interference. SYNGAP1-AS1 is expressed in the human brain, with region-specific enrichment, and is upregulated in the prefrontal cortex and superior temporal gyrus of individuals with autism spectrum disorder compared to controls[2]. The negative correlation between SYNGAP1-AS1 and SYNGAP1, and its potential to be targeted by antisense oligonucleotide therapies to restore SYNGAP1 levels, establish it as a novel therapeutic target for neurodevelopmental conditions, particularly SYNGAP1 haploinsufficiency syndromes[3][6]. The intervention strategy centers on reducing SYNGAP1-AS1 activity to upregulate the beneficial SYNGAP1 gene product in affected neuronal populations.

Other names
SYNGAP1-ASSYNGAP1 natural antisense transcriptSYNGAP1-AS1 RNA
02

Mechanism of action

Antisense oligonucleotides bind to SYNGAP1-AS1 to inhibit or modulate its function, leading to increased SYNGAP1 mRNA and protein expression[3][4][6].

03

Biological functions

Epigenetic regulationNegative regulation of SYNGAP1 gene expressionModulation of synaptic protein levels
04

Disease associations

Autism spectrum disorderIntellectual disability
05

Safety considerations

Delivery of antisense oligonucleotides to the brain (requires intrathecal or other CNS-targeted delivery)Off-target modulation of other genes or unintended epigenetic changes[3]
06

Interacting drugs

Antisense oligonucleotides (experimental; no approved drugs listed by name)

Beyond the preview

Go deeper on SYNGAP1 antisense RNA 1 (SYNGAP1-AS1).

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

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

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

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

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 SYNGAP1 antisense RNA 1 (SYNGAP1-AS1).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call