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Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing.
Kaiser RA, Carlson DF, Allen KL, Webster DA, VanLith CJ, Nicolas CT, Hillin LG, Yu Y, Kaiser CW, Wahoff WR, Hickey RD, Watson AL, Winn SR, Thöny B, Kern DR, Harding CO, Lillegard JB. Kaiser RA, et al. Among authors: vanlith cj. PLoS One. 2021 Jan 25;16(1):e0245831. doi: 10.1371/journal.pone.0245831. eCollection 2021. PLoS One. 2021. PMID: 33493163 Free PMC article.
Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure.
Nicolas CT, Kaiser RA, Hickey RD, Allen KL, Du Z, VanLith CJ, Guthman RM, Amiot B, Suksanpaisan L, Han B, Francipane MG, Cheikhi A, Jiang H, Bansal A, Pandey MK, Garg I, Lowe V, Bhagwate A, O'Brien D, Kocher JA, DeGrado TR, Nyberg SL, Lagasse E, Lillegard JB. Nicolas CT, et al. Among authors: vanlith cj. Mol Ther Methods Clin Dev. 2020 Jul 10;18:738-750. doi: 10.1016/j.omtm.2020.07.009. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32913881 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: vanlith cj. Nat Commun. 2022 Aug 25;13(1):5012. doi: 10.1038/s41467-022-32576-7. Nat Commun. 2022. PMID: 36008405 Free PMC article.
13 results