SMN2 antisense RNA 1 (SMN2-AS1)

SMN2 antisense RNA 1 is a long non-coding RNA transcribed from the SMN locus that plays an epigenetic regulatory role by binding to the SMN transcription start site region and recruiting Polycomb Repressive Complex 2 (PRC2), leading to reduced transcription of the SMN2 gene. Experimental studies have shown that inhibiting the interaction between SMN2-AS1 and PRC2 with chemically modified antisense oligonucleotides upregulates SMN2 expression in neuronal cells, providing a novel therapeutic strategy for spinal muscular atrophy (SMA). SMN2-AS1 does not code for a protein and is not itself a receptor, enzyme, or transporter, but is a regulatory RNA molecule involved in modulating the epigenetic silencing of SMN2, thus affecting SMN protein levels in the context of SMA.

Other names

SMN2-AS1SMN-AS1SMN-antisense 1

Antisense oligonucleotides block SMN2-AS1 interaction with PRC2, resulting in reduced recruitment of PRC2 and derepression (upregulation) of SMN2 transcription