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Lori G. Hillin

Mayo Clinic

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Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing

2021 PLOS ONE
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Use of an AAV serotype Anc80 to provide durable cure of phenylketonuria in a mouse model.

2021 Journal of Inherited Metabolic Disease
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In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

2021 bioRxiv
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