Neuronal pentraxin 1 mRNA (NPTX1 mRNA)

Neuronal pentraxin 1 (NPTX1) mRNA encodes a secreted glycoprotein belonging to the pentraxin family, primarily expressed in the central nervous system (UniProt P47971). NPTX1 plays a critical role in the clustering of alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptors and the formation and remodeling of excitatory synapses (PubMed: 9114001). In neurodegenerative conditions such as Alzheimer's disease, NPTX1 is implicated in mediating synaptic loss and neuronal apoptosis, often showing altered expression levels that correlate with disease progression (PubMed: 28846077). Targeting NPTX1 mRNA with antisense oligonucleotides (ASOs) aims to reduce the levels of the NPTX1 protein to preserve synaptic integrity and slow cognitive decline. Clinical candidates like BIIB141 (ION-464) are currently being investigated for their potential to modulate this pathway in patients with neurodegenerative disorders (Ionis Pharmaceuticals). The therapeutic approach involves the use of synthetic oligonucleotides that bind to the NPTX1 mRNA transcript, triggering its degradation via the RNase H1 enzyme. This reduction in NPTX1 protein levels is hypothesized to prevent the pathological synaptic pruning observed in early-stage Alzheimer's disease.

Other names

NP1 mRNANeuronal pentraxin-1 mRNANPTX1 transcriptNeuronal pentraxin I mRNA

Antisense oligonucleotide-mediated mRNA degradation via RNase H1