Article
The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis.
Science translational medicine - 24 May 2017
Imamura Keiko, Izumi Yuishin, Watanabe Akira, Tsukita Kayoko, Woltjen Knut, Yamamoto Takuya, Hotta Akitsu, Kondo Takayuki, Kitaoka Shiho, Ohta Akira, Tanaka Akito, Watanabe Dai, Morita Mitsuya, Takuma Hiroshi, Tamaoka Akira, Kunath Tilo, Wray Selina, Furuya Hirokazu, Era Takumi, Makioka Kouki, Okamoto Koichi, Fujisawa Takao, Nishitoh Hideki, Homma Kengo, Ichijo Hidenori, Julien Jean-Pierre, Obata Nanako, Hosokawa Masato, Akiyama Haruhiko, Kaneko Satoshi, Ayaki Takashi, Ito Hidefumi, Kaji Ryuji, Takahashi Ryosuke, Yamanaka Shinya, Inoue Haruhisa
Abstract excerpt
Amyotrophic lateral sclerosis (ALS), a fatal disease causing progressive loss of motor neurons, still has no effective treatment. We developed a phenotypic screen to repurpose existing drugs using ALS motor neuron survival as readout. Motor neurons were generated from induced pluripotent stem cells (iPSCs) derived from an ALS patient with a mutation in superoxide dismutase 1 (SOD1). Results of the screen showed...
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