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Correction of mutant Fanconi anemia gene by homologous recombination in human hematopoietic cells using adeno‐associated virus vector

Adeno‐associated virus (AAV) vectors have been shown to correct a variety of mutations in human cells by homologous recombination (HR) at high rates, which can overcome insertional mutagenesis and transgene silencing, two of the major hurdles in conventional gene addition therapy of inherited diseases. We examined an ability of AAV vectors to repair a mutation in human hematopoietic cells by HR.