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Clinical evidences supporting the Src/c-Abl pathway as potential therapeutic target in amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS), the most common degenerative disease affecting motor neurons, lacks an effective disease-modifying disease treatment [1–3]. Recently, the Src/c-Abl pathway has been postulated as a potential therapeutic target in ALS. [4]. Imamura et al. developed a phenotypic screen to repurpose existing drugs for ALS patients, which resulted in the identification of 27 potential hits. Intriguingly, more than half of the hits targeted the Src/c-Abl signaling pathway [4]. Complementary studies demonstrated that Src/c-Abl inhibitors increased the survival of induced pluripotent stem cells (iPSC)-derived motor neurons from ALS patients in vitro.

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