Kumamoto University

Shiro Matsumoto

Fumio Endo

Takumi Era

[Object] To clarify the mechanism associated with neuropathy in methylmalonic acidemia and to develop a new therapeutic drug or the like for neuropathy in organic acidemia on the basis of this finding. [Solving Means] The inventors established technologies for the establishment of iPS cells derived from a methylmalonic acidemia patient and establishment of a stable maintenance and culturing method using peripheral blood lymphocytes of a methylmalonic acidemia patient, and for the differentiation of methylmalonic acidemia patient-derived iPS cells into nerve cells. The inventors made clear that neuropathy in organic acidemia can be treated and prevented by replenishing cAMP using a series of these experiment technologies. The drug of the invention treats or prevents neuropathy by increasing cAMP in organic acidemia.

Growing community of inventors

Kumamoto, Japan

Shiro Matsumoto