Antisense Oligonucleotide Therapeutic Approach for Suppression of Ataxin-1 Expression: A Safety Assessment.

Mol Ther Nucleic Acids · 2020 · PMC7452125 · PMID 32818920

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highly significantno p-value reported

In dramatic contrast, Atxn1 154Q/2Q mice receiving injections of the non-targeting ASO showed a highly significant decline in motor performance from 5 to 25 weeks.

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