CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I.

Nat Commun · 2018 · PMC6303323 · PMID 30575740

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highly significantno p-value reported

We have demonstrated that a sustained and highly significant reduction of GO protein expression can be achieved after a single delivery of an AAV vector carrying both Staphylococcus aureus Cas9 and GO-specific sgRNAs to the livers of mice with PH1.

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