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Regulatory RNA of SYNGAP1 gene (None established; if specific regulatory RNAs are identified, names such as SYNGAP1-AS1 (for antisense RNA) are sometimes used[6].)

Target
None established; if specific regulatory RNAs are identified, names such as SYNGAP1-AS1 (for antisense RNA) are sometimes used[6].
Molecular classification
Long non-coding RNA (lncRNA)[6], Natural antisense transcript (NAT)[3], Enhancer RNA (eRNA)[3], Promoter-associated RNA (paRNA)[3], Other non-coding RNA
01

Overview

Regulatory RNAs of the SYNGAP1 gene encompass a variety of non-coding RNA molecules, including long non-coding RNAs, antisense RNAs, enhancer RNAs, and promoter-associated RNAs, which modulate the levels and activity of SYNGAP1 mRNA and protein in neurons[3][6]. These RNAs act by influencing transcription, alternative splicing, mRNA stability, and decay, shaping the amount of SYNGAP1 protein available, thereby impacting synaptic development, plasticity, and cognitive function[5][6][7]. Alterations, mutations, or aberrant regulation of these RNAs contribute to neurodevelopmental diseases, and RNA-based therapies (such as antisense oligonucleotides) targeting these regulatory elements are being developed to restore normal SYNGAP1 function and treat associated disorders[3][5][7]. Because this entry refers collectively to several regulatory RNA types rather than one molecule, it is not an ideal structured target designation, and the specific RNA (e.g., SYNGAP1-AS1) should be named where possible for clarity[6].

Other names
SYNGAP1 regulatory RNASYNGAP1-AS1 (for the antisense RNA)[6]SYNGAP1 non-coding RNA
02

Mechanism of action

Upregulation of SYNGAP1 expression by blocking unproductive splicing or nonsense-mediated decay of SYNGAP1 mRNA[5][7] Modulation of regulatory RNA stability or expression to alter SYNGAP1 gene output[3][5][7]

03

Biological functions

Regulation of gene expression[3][6]Modulation of mRNA splicing[5][7]Control of mRNA stability and decay (nonsense-mediated decay)[5][7]Synaptic plasticity (indirectly, through SYNGAP1 protein)[5][6]Brain development (indirectly)[5][6]
04

Disease associations

Neurodevelopmental disorders (including intellectual disability, autism spectrum disorder, epilepsy)[1][2][5][6][7]Specific learning disorder[6]Other (potentially other brain or cognitive disorders)[3][8]
05

Safety considerations

Off-target effects of oligonucleotide therapies[3][5]Potential for immune activation by nucleic acid drugs[3]Unintended alteration of neuronal or synaptic functions causing seizures, cognitive impairment, or neuropsychiatric symptoms[5][6][7]
06

Interacting drugs

Antisense oligonucleotides (ASOs) targeting SYNGAP1 regulatory RNAs[3]

1 more in the full profile.

07

Biomarkers

Expression levels of SYNGAP1 regulatory RNAs (such as SYNGAP1-AS1)[6]SYNGAP1 mRNA or protein levels[6][5]Alternative splicing patterns of SYNGAP1 transcript[5][7]

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