News Releases & Research Results Abnormalities in neurons differentiated from iPS cells derived from patients with familial dementia were detected―Development of a new therapeutic agent for diseases caused by mutations in the protein tau is expected―
News Releases & Research Results
Results of R&D by a research group of Professor Hideyuki Okano and Visiting Associate Researcher Mari Nakamura of the Department of Physiology, School of Medicine, Keio University, Specially Appointed Instructor Seiji Shiozawa of the Center for Integrated Medical Research, School of Medicine, Keio University, and others.
Key points of the study results
- iPS cells were established from patients with familial frontotemporal lobar degeneration (FTLD: a type of dementia that occurs due to neuronal death in the frontal lobe in old age), and part of the disease mechanism was elucidated by using differentiated neurons.
- Specifically, in iPS cells derived from neurons with mutations in the microtubule-associated protein tau gene (the R406W tau mutation), which is one of the genes that cause FTLD, phosphorylation and anomalous localization of protein tau, axonal degeneration, etc. were observed. In addition, these abnormalities were found to be suppressed by a microtubule stabilizer.
- The study results are expected to help the development of therapeutic agents for diseases caused by the protein tau or drugs that can control the progression of diseases.
This R&D was conducted with the support of Research Center Network Program for Realization of Regenerative Medicine by AMED.
The results of this R&D were published on September 19 in the online version of Stem Cell Reports, an official journal of the International Society for Stem Cell Research (ISSCR).
Nakamura M., et al. Pathological progression induced by the frontotemporal dementia-associated R406W tau mutation in patient-derived iPSCs, Stem Cell Reports
Last updated 2019.9.20