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Description

Gene therapy

Constitutive activation of -catenin in neural progenitors results in disrupted proliferation and migration of neurons within the central nervous system

Targeted experimental validation was performed in murine myotubes/muscle tissue from mALD for mitochondrial oxidative function using high sensitivity respirofluorometry, structural evaluation of mitochondrial morphology using confocal microscopy, cristae morphology on 3D electron microscopy, HIF1a, and myostatin expression by immunoblots