Aug 2026
MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse model.
Treatment with splicing modulator compounds that promote MAPT exon 10 exclusion reduced 4R tau expression and decreased the accumulation of hyperphosphorylated tau (pTau) and oligomeric and insoluble tau proteoforms, thereby rescuing tau-associated neuronal toxicity.
M. Silva, Hannah Lindmeier, Paolo Pigini et al.
· Science Translational Medici... · 1 citation
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