Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Natural antisense transcript of sodium channel protein type 1 subunit alpha
- Molecular classification
- Long non-coding RNA (lncRNA), Natural antisense transcript, Regulatory RNA
01
Overview
The natural antisense transcript of sodium channel protein type 1 subunit alpha is a long non-coding RNA molecule generated from the DNA opposite the SCN1A gene, which codes for a voltage-gated sodium channel critical for action potential initiation in neurons. This antisense RNA regulates the amount of SCN1A mRNA and protein, functioning as a gene expression modulator. Therapeutic strategies targeting this NAT, such as antisense oligonucleotides, are under investigation for disorders like Dravet syndrome and chronic pain conditions, where altered SCN1A expression contributes to pathology. Antisense manipulation provides a way to treat haploinsufficiency or gain-of-function disease states by restoring or controlling protein levels.
- Other names
- SCN1A-NATSCN1A natural antisense transcriptSCN1A antisense RNANAT of sodium channel protein type 1 subunit alpha
02
Mechanism of action
Gene expression modulation via ASOs that interfere with the natural antisense transcript, leading to increased productive SCN1A mRNA and protein expression or knockdown of target gene expression by silencing SCN1A-NAT
03
Biological functions
Regulation of SCN1A gene expressionModulation of neuronal excitabilityRegulation of mRNA and protein levels of sodium channel protein type 1 subunit alpha
04
Disease associations
Epilepsy (specifically Dravet syndrome)Chronic pain syndromes (e.g., Inherited Primary Erythromelalgia, Paroxysmal Extreme Pain Disorder, Painful Small Fibre Neuropathy—when SCN1A is involved)
05
Safety considerations
Off-target effects of ASOsPotential for immune activationRisk of unintended neuronal excitability changes causing seizures
06
Interacting drugs
Antisense oligonucleotides (ASOs) designed to either increase or decrease SCN1A protein by modulating NAT levels
07
Biomarkers
Levels of productive SCN1A mRNASCN1A protein expressionSeizure frequency and severity (in clinical research epilepsy models)
Beyond the preview
Go deeper on Natural antisense transcript of sodium channel protein type 1 subunit alpha.
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 Natural antisense transcript of sodium channel protein type 1 subunit alpha.