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Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1.
Hickey RD, Mao SA, Glorioso J, Elgilani F, Amiot B, Chen H, Rinaldo P, Marler R, Jiang H, DeGrado TR, Suksanpaisan L, O'Connor MK, Freeman BL, Ibrahim SH, Peng KW, Harding CO, Ho CS, Grompe M, Ikeda Y, Lillegard JB, Russell SJ, Nyberg SL. Hickey RD, et al. Sci Transl Med. 2016 Jul 27;8(349):349ra99. doi: 10.1126/scitranslmed.aaf3838. Sci Transl Med. 2016. PMID: 27464750 Free PMC article.
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions.
Nicolas CT, VanLith CJ, Hickey RD, Du Z, Hillin LG, Guthman RM, Cao WJ, Haugo B, Lillegard A, Roy D, Bhagwate A, O'Brien D, Kocher JP, Kaiser RA, Russell SJ, Lillegard JB. Nicolas CT, et al. Among authors: hickey rd. Nat Commun. 2022 Aug 25;13(1):5012. doi: 10.1038/s41467-022-32576-7. Nat Commun. 2022. PMID: 36008405 Free PMC article.
Limited Expansion of Human Hepatocytes in FAH/RAG2-Deficient Swine.
Nelson ED, Larson E, Joo DJ, Mao S, Glorioso J, Abu Rmilah A, Zhou W, Jia Y, Mounajjed T, Shi M, Bois M, Wood A, Jin F, Whitworth K, Wells K, Spate A, Samuel M, Minshew A, Walters E, Rinaldo P, Lillegard JB, Johnson A, Amiot B, Hickey R, Prather R, Platt JL, Nyberg SL. Nelson ED, et al. Tissue Eng Part A. 2022 Feb;28(3-4):150-160. doi: 10.1089/ten.TEA.2021.0057. Epub 2021 Oct 7. Tissue Eng Part A. 2022. PMID: 34309416 Free PMC article.
29 results