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Rett Syndrome and Stem Cell Research

  • Keita Tsujimura
  • Kinichi Nakashima
Chapter

Abstract

Rett syndrome (RTT) is a devastating neurodevelopmental disorder resulting from mutations in the X-linked gene encoding methyl-CpG-binding protein 2 (MECP2). MECP2 mutations are also associated with other neurodevelopmental diseases, including autism and schizophrenia. Therefore, elucidating the mechanism of RTT can contribute to understanding the pathogenesis of a wide range of neurodevelopmental diseases. Despite its importance, however, the RTT pathogenesis remains unclear, and effective therapeutic treatment has not been developed. Offering an opportunity to move toward this goal, however, is the recent advance in the stem cell research field of the development of induced pluripotent stem cell (iPSC) technology. This technology can yield important insights into disease pathophysiology and has the potential to provide disease models for screening new drugs. Here, we discuss applications of recent stem cell technology to the field of research on RTT and describe the stem cell biology of RTT pathogenesis.

Keywords

MeCP2 Rett syndrome Neurodevelopmental disorders Induced pluripotent stem cells (iPSCs) Neural stem cells (NSCs) 

Notes

Acknowledgments

This study was in part supported by JSPS KAKENHI Grant Number 16 K18391 to K.T. and MEXT KAKENHI Grant Number 17H01390 to K.N., Foundation of Synapse and Neurocircuit Pathology, and Intramural Research Grant 27-7 for Neurological and Psychiatric Disorders of the National Center of Neurology and Psychiatry. We thank Elizabeth Nakajima for critical reading of the manuscript.

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Authors and Affiliations

  1. 1.Department of PsychiatryNagoya University Graduate School of MedicineShowa, NagoyaJapan
  2. 2.Institute for Advanced Research, Nagoya UniversityChikusa, NagoyaJapan
  3. 3.Department of Stem Cell Biology and MedicineGraduate School of Medical Sciences, Kyushu UniversityHigashi-ku, FukuokaJapan

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