Discovery of a New Candidate Factor Associated with the Pathogenesis of Amyotrophic Lateral Sclerosis (ALS)—Toward Elucidating the Mechanism of Selective Motor Neuron Degeneration Using Patient-Derived iPS Cells—

The expression of the PHOX2B gene was found to be decreased in motor neurons derived from human iPS cells established from patients with familial amyotrophic lateral sclerosis.

Suppression of PHOX2B expression in a model animal (zebrafish) shortened neurite length and reduced motor function.

The PHOX2B gene and its related molecules were shown to be potential new therapeutic targets for amyotrophic lateral sclerosis.