New from CGM in Science Translational Medicine: CGM's Silva, Morini & team developed small molecules that fix a MAPT splicing error, lowering toxic 4R tau in FTD neurons and a mouse tauopathy model. A promising step toward FTD-tau therapeutics. doi.org/10.1126/scitranslmed.ady6759
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MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse model
MAPT splicing modulator compounds reduce 4R tau and rescue pathology in neurons derived from patients with tauopathy and mouse models.
