Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡

Abstract excerpt

UNLABELLED: Adeno-associated virus (AAV) vectors are ideal for performing gene repair due to their ability to target multiple different genomic loci, low immunogenicity, capability to achieve targeted and stable expression through integration, and low mutagenic and oncogenic potential. However, many handicaps to gene repair therapy remain. Most notable is the low frequency of correction in vivo. To date, this...

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