Hitoshi Warita

Intractable neurological diseases became an increasingly worrisome burden for our aging population in Japan. We are currently focusing on amyotrophic lateral sclerosis (ALS) and neuromuscular diseases including rare and intractable myopathies.
Most of the neurodegenerative diseases such as ALS are characterized by late-onset, progressive dysfunction and loss of neuronal subpopulations that affect a specific neural system. Currently available treatments are, however, critically limited.
The identification of disease-causing genes in their familial forms has offered substantial insights into the molecular pathomechanisms underlying the diseases. Thus, we employ next-generation sequencing to find novel disease-related genes. Moreover, a variety of animal and cellular models including patient-derived induced pluripotent stem cells (iPSC) have been developed to clarify the pathomechanisms, leading to clinical translation.