Clinical utility of neuronal cells directly converted from fibroblasts of patients for neuropsychiatric disorders: studies of lysosomal storage diseases and channelopathy.

@article{Kano2015ClinicalUO,
  title={Clinical utility of neuronal cells directly converted from fibroblasts of patients for neuropsychiatric disorders: studies of lysosomal storage diseases and channelopathy.},
  author={S-i Kano and Meng Yuan and Ross A Cardarelli and Gustavo H B Maegawa and Norimichi Higurashi and Meriem Gaval-Cruz and Ashley M. Wilson and Cenier Tristan and Mari A. Kondo and Yu-chi Chen and Minori Koga and Cassandra Obie and Kanako Ishizuka and Sriram Seshadri and Rupali Srivastava and Takahiro A Kato and Yoshitaka Horiuchi and Thomas W. Sedlak and Y.-H. Lee and Judith L. Rapoport and Shigeto Hirose and Hideyuki Okano and David Valle and Patricio O'Donnell and Akira Sawa and Mihoko Kai},
  journal={Current molecular medicine},
  year={2015},
  volume={15 2},
  pages={138-45}
}
Methodologies for generating functional neuronal cells directly from human fibroblasts [induced neuronal (iN) cells] have been recently developed, but the research so far has only focused on technical refinements or recapitulation of known pathological phenotypes. A critical question is whether this novel technology will contribute to elucidation of novel disease mechanisms or evaluation of therapeutic strategies. Here we have addressed this question by studying Tay-Sachs disease, a… CONTINUE READING
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