Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.

J Inherit Metab Dis · 2021 · PMC9291745 · PMID 33896013

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showed a trendno p-value reported

Trends were similar to VG results, whereby both AAVs showed similar PAH mRNA levels at the 5 × 10 11 VG/kg dose, but Anc80‐coPAH showed a trend for slightly better transcription than Anc80‐int‐coPAH at the 5 × 10 12 VG/kg dose.

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