A potential therapeutic approach for tauopathies

Abstract

A pathological hallmark of several neurodegenerative disorders is the accumulation of misfolded protein aggregates. For instance, the accumulation of misfolded and hyperphosphorylated microtubule-associated protein tau (MAPT, tau) is the hallmark of tauopathies. Because tau pathology might vary between disorders, a pragmatic therapeutic approach may be to target the tau transcript, which makes no assumptions about tau toxicity. In this issue of Molecular Therapy: Nucleic Acids, Amy Easton et al.