FMR1 antisense RNA 1 (FMR1-AS1)

FMR1 antisense RNA 1 (FMR1-AS1, also called ASFMR1 or FMR4) is a long non-coding RNA gene overlapping and antisense to the CGG repeat region of the FMR1 gene. It is upregulated in carriers of the Fragile X premutation and silenced in full mutation Fragile X syndrome. FMR1-AS1 exhibits alternative splicing, cytoplasmic export, and is expressed predominantly in the brain and kidney. Its functions include epigenetic regulation of FMR1 gene expression, neuroprotective and anti-apoptotic effects (by regulating apoptosis, oxidative stress, and mTOR/Tau signaling). It may contribute to disease pathology or protection in neurodevelopmental disorders, Alzheimer’s disease, and some cancers, pointing to its emerging therapeutic and biomarker potential. This molecule is a regulatory RNA, not a classical drug receptor, enzyme, or transporter, but it represents a novel class of ncRNA-based therapeutic targets and biomarkers in neurodegeneration and epigenetic disorders.

Other names

ASFMR1FMR4FMR5antisense fragile X messenger ribonucleoprotein 1antisense fragile X mental retardation proteinFMR1-ASFMR1ASFMR1 antisense RNA 1 (head to head)FMR1 antisense RNA 1 (non-protein coding)

For future therapies: Regulation of FMR1-AS1 expression may modulate apoptosis and synaptic function via epigenetic and translational control mechanisms. Modulation of TLR7–NF-κB signaling pathway. mTOR pathway regulation (relevant to synaptic plasticity).