Tau-targeting antisense oligonucleotide

Tau-targeting antisense oligonucleotides (ASOs) are a class of RNA-targeted therapeutics designed to bind to microtubule-associated protein tau (MAPT) pre-mRNA or mRNA, leading to its degradation (typically via RNase H1 recruitment) and subsequently reducing the production of all isoforms of the tau protein. By lowering both intracellular and extracellular tau levels, these therapies aim to prevent the misfolding, aggregation, and accumulation of hyperphosphorylated tau into neurofibrillary tangles, which are key pathological hallmarks of Alzheimer's disease and other primary tauopathies (such as progressive supranuclear palsy). Several candidates in this class, such as diranersen (BIIB080) and NIO752, are undergoing clinical evaluation to assess their ability to slow cognitive and functional decline in neurodegenerative disorders.

Other names

MAPT ASOMAPT antisense oligonucleotideMAPT-targeting antisense oligonucleotidetau ASOtau-targeting ASO