Helen C. Miranda

Emerin loss of function inhibits MyoD-driven differentiation of human iPSCs into skeletal myotubes

This study shows that emerin loss impairs MyoD-driven differentiation of human iPSCs into skeletal myotubes by affecting PRC2-mediated gene regulation.

Mitochondrial dysfunction heightens the integrated stress response to drive ALS pathogenesis

This study shows that VAPB P56S mutation causes mitochondrial dysfunction and enhances the integrated stress response, leading to motor neuron disease in ALS.

Scalable, optically-responsive human neuromuscular junction model reveals convergent mechanisms of synaptic dysfunction in familial ALS

This study develops a human neuromuscular junction model from pluripotent stem cells to investigate synaptic dysfunction in familial ALS and assess therapeutic candidates.

Skeletal Muscle TFEB Signaling Promotes Central Nervous System Function and Reduces Neuroinflammation during Aging and Neurodegenerative Disease

This study shows that enhancing skeletal muscle TFEB signaling reduces neuroinflammation and tau pathology in aging and neurodegenerative disease models.