In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions
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DOI: 10.1038/s41467-022-32576-7
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- Francis P. Pankowicz & Mercedes Barzi & Xavier Legras & Leroy Hubert & Tian Mi & Julie A. Tomolonis & Milan Ravishankar & Qin Sun & Diane Yang & Malgorzata Borowiak & Pavel Sumazin & Sarah H. Elsea & , 2016. "Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia," Nature Communications, Nature, vol. 7(1), pages 1-6, November.
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