Alpha-synuclein messenger RNA (SNCA mRNA) (SNCA mRNA)

Alpha-synuclein messenger RNA (SNCA mRNA) is the primary transcript of the SNCA gene, which encodes the alpha-synuclein protein, a critical component of the presynaptic terminal involved in vesicle trafficking and neurotransmitter release (NCBI Gene, 2024; Stefanis, 2012). In neurodegenerative conditions known as synucleinopathies, including Parkinson's disease and Multiple System Atrophy, the overproduction or mutation of this protein leads to the formation of toxic aggregates called Lewy bodies (Stefanis, 2012). Because the pathology is closely linked to the concentration of alpha-synuclein, targeting the mRNA to reduce protein synthesis has emerged as a primary therapeutic strategy (Cole et al., 2021). Current drug candidates, such as antisense oligonucleotides (ASOs) like BIIB094, are designed to bind to SNCA mRNA and trigger its degradation via RNase H, thereby lowering the total protein burden in the brain (ClinicalTrials.gov, 2023; Cole et al., 2021). This approach aims to modify the disease course by preventing the initial steps of protein aggregation and subsequent neuronal death. By reducing the expression of both wild-type and mutant forms of the protein, these therapies offer a potential disease-modifying treatment for patients with progressive neurodegeneration.

Other names

Alpha-synuclein mRNASNCA transcriptNACP mRNAPARK1 mRNAPARK4 mRNA

Antisense oligonucleotide-mediated RNase H degradation and RNA interference (RNAi) to reduce protein translation.