Acetylcholinesterase mRNA (AChE mRNA) (AChE mRNA)

Acetylcholinesterase (AChE) mRNA is the messenger RNA transcript that encodes the enzyme responsible for the rapid hydrolysis of acetylcholine at cholinergic synapses (Soreq & Seidman, 2001, Nature Reviews Neuroscience). Under conditions of physiological stress or chronic disease, the ACHE gene undergoes alternative splicing, leading to the accumulation of the AChE-R (read-through) mRNA isoform, which is associated with pathological cholinergic signaling and neuroinflammation (Evron et al., 2007, Journal of Molecular Neuroscience). Therapeutic targeting of AChE mRNA, primarily through antisense oligonucleotides (ASOs) like Monarsen (EN101), aims to selectively degrade these transcripts or block their translation to reduce enzyme levels (Sussman et al., 2008, Journal of Neurochemistry). This approach is particularly relevant for treating Myasthenia gravis, where reducing AChE levels improves neuromuscular transmission, and has been explored for Alzheimer's disease and organophosphate poisoning (Brenner et al., 2003, FASEB Journal). By targeting the mRNA rather than the protein, these therapies offer a way to modulate specific splice variants and achieve more sustained pharmacological effects than traditional small-molecule inhibitors (Nadorp & Soreq, 2014, Frontiers in Molecular Neuroscience). This strategy represents a precision medicine approach to managing disorders of the cholinergic system by addressing the underlying transcriptomic dysregulation.

Other names

ACHE messenger RNAAcetylcholinesterase transcriptAChE-R mRNAAChE-S mRNAAChE-H mRNA

Antisense oligonucleotide-mediated degradation of mRNA via RNase H or translational blockade to reduce the production of acetylcholinesterase protein (Sussman et al., 2008, Journal of Neurochemistry).