<i>In vivo</i> CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluria.

We have demonstrated that a single dose of an AAV8 vector carrying the SaCas9 and a sgRNA targeting LDH, administered into the livers of Agxt1 −/− and Hoga1 −/− mice, models of PH1 and PH3, respectively, efficiently and specifically induced indels in the target gene ( Ldha ), resulting in a sustained and highly significant reduction of LDH protein.

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