Research ArticleAmyotrophic Lateral Sclerosis

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

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Science Translational Medicine  31 Oct 2018:
Vol. 10, Issue 465, eaau6414
DOI: 10.1126/scitranslmed.aau6414

A silent attack on ALS

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder for which there are currently no available therapies. Gain-of-function mutations in the gene encoding superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS cases. Silencing SOD1 using artificial microRNA has been shown to have therapeutic effects in mouse models of ALS. Here, Borel et al. tested the efficacy and safety of intrathecal delivery of SOD1-targeting artificial microRNAs in nonhuman primates. The therapy efficiently reduced SOD1 protein expression without side effects in macaques. The results suggest that this approach is safe and effective, paving the way for further development of this potential therapy.

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