National Center for Neurology and Phychiatry Department of Molecular Therapy, National Institute of Neuroscience

Research outline

The Department of Molecular Therapy is dedicated to the elucidation of molecular pathogenesis of muscular dystrophy and the development of advanced treatment for the disease. Among various muscular dystrophies, Duchenne muscular dystrophy (DMD) is a devastating muscle disorder caused by mutations in the dystrophin gene and there is currently no established treatment. We are tackling this disease through the researches aiming to bring the promising novel therapeutic strategies, including oligonucleotide-based exon-skipping therapy and induced pluripotent stem cell (iPSC)-based cell therapy, into clinical practice. We are also studying genetics, pathogensis and clinical interventions in DMD using muscular dystrophy dogs, which are genuine models of human DMD. Furthermore, to elucidate the complex molecular mechanisms of muscular dystrophy, we are focusing on the functional analysis of intracellular calcium regulation and mechanosensing in muscle cells.

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September. 26. 2017
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July. 20. 2016
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