Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent

• Published in: Nature medicine Vol. 24; no. 10; pp. 1579 - 1589
• Main Authors: Fujimori, Koki, Ishikawa, Mitsuru, Otomo, Asako, Atsuta, Naoki, Nakamura, Ryoichi, Akiyama, Tetsuya, Hadano, Shinji, Aoki, Masashi, Saya, Hideyuki, Sobue, Gen, Okano, Hideyuki
• Format: Journal Article
• Language: English
• Published: United States Nature Publishing Group 01.10.2018
• Subjects: Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - genetics; Amyotrophic Lateral Sclerosis - pathology; Cell death; Cell Differentiation - genetics; Chemical compounds; Clustering; Degeneration; Drug development; Humans; Indoles - therapeutic use; Induced Pluripotent Stem Cells - metabolism; Modelling; Motor neuron diseases; Motor Neurons - metabolism; Motor Neurons - pathology; Motors; Mutation; Nerve Degeneration - genetics; Nerve Degeneration - metabolism; Nerve Degeneration - pathology; Pathogenesis; Patients; Pharmacology; Phenotype; Phenotypes; Pluripotency; Protein Aggregation, Pathological - genetics; Proteins; Stem cells; Superoxide dismutase; Superoxide Dismutase-1 - genetics

Amyotrophic lateral sclerosis (ALS) is a heterogeneous motor neuron disease for which no effective treatment is available, despite decades of research into SOD1-mutant familial ALS (FALS). The majority of ALS patients have no familial history, making the modeling of sporadic ALS (SALS) essential to...