Microtubule-associated protein tau (MAPT) mRNA (MAPT mRNA)

Microtubule-associated protein tau (MAPT) mRNA is the transcript responsible for the synthesis of tau protein, which is essential for stabilizing neuronal microtubules (UniProt P10636). In neurodegenerative diseases collectively termed tauopathies, such as Alzheimer's disease and Progressive Supranuclear Palsy, tau protein aggregates into toxic neurofibrillary tangles (PubMed: 29926062). Targeting the MAPT mRNA transcript using antisense oligonucleotides (ASOs) like BIIB080 or RNA interference (RNAi) molecules like ALN-MAPT allows for the reduction of tau protein production at the source (ClinicalTrials.gov: NCT03186911). This strategy aims to decrease the concentration of all tau isoforms, potentially halting the progression of tau-mediated neurodegeneration and preserving cognitive function (PubMed: 31511698). Current clinical efforts focus on the safety and efficacy of lowering tau levels in the central nervous system to treat both familial and sporadic tauopathies. This approach represents a significant shift from targeting extracellular protein aggregates to addressing the intracellular production of the pathogenic protein itself.

Other names

Tau mRNAMAPT transcriptMicrotubule-associated protein tau transcript

Antisense oligonucleotide-mediated RNase H degradation or RNA interference (RNAi) to reduce protein expression