Article
Directly converted patient-specific induced neurons mirror the neuropathology of FUS with disrupted nuclear localization in amyotrophic lateral sclerosis.
Molecular neurodegeneration - 22 Jan 2016
Lim Su Min, Choi Won Jun, Oh Ki-Wook, Xue Yuanchao, Choi Ji Young, Kim Sung Hoon, Nahm Minyeop, Kim Young-Eun, Lee Jinhyuk, Noh Min-Young, Lee Seungbok, Hwang Sejin, Ki Chang-Seok, Fu Xiang-Dong, Kim Seung Hyun
Abstract excerpt
BACKGROUND: Mutations in the fused in sarcoma (FUS) gene have been linked to amyotrophic lateral sclerosis (ALS). ALS patients with FUS mutations exhibit neuronal cytoplasmic mislocalization of the mutant FUS protein. ALS patients' fibroblasts or induced pluripotent stem cell (iPSC)-derived neurons have been developed as models for understanding ALS-associated FUS (ALS-FUS) pathology; however, pathological...
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