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Article

Genetic validation of a therapeutic target in a mouse model of ALS.

Science translational medicine - 6 Aug 2014

de Boer A Sophie, Koszka Kathryn, Kiskinis Evangelos, Suzuki Naoki, Davis-Dusenbery Brandi N, Eggan Kevin

Abstract excerpt

Neurons produced from stem cells have emerged as a tool to identify new therapeutic targets for neurological diseases such as amyotrophic lateral sclerosis (ALS). However, it remains unclear to what extent these new mechanistic insights will translate to animal models, an important step in the validation of new targets. Previously, we found that glia from mice carrying the SOD1G93A mutation, a model of ALS, were...

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PMID: 25100738
DOI: 10.1126/scitranslmed.3009351

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