Gaj T, Ojala DS, Ekman FK, Byrne LC, Limsirichai P, Schaffer DV. In vivo genome editing improves motor function and extends survival in a mouse model of ALS. Sci Adv. 2017 Dec;3(12):eaar3952. Epub 2017 Dec 20 PubMed.

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  1. This is an extremely important, albeit not entirely surprising, observation given the link between mutated forms of this enzyme and familial ALS. Unfortunately, gene editing is not likely to be the "Holy Grail" for persons diagnosed with sporadic ALS.

    View all comments by Marcia Ratner

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