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