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Genetic therapy in a mitochondrial disease model suggests a critical role for liver dysfunction in mortality.
Sabharwal A, Wishman MD, Cervera RL, Serres MR, Anderson JL, Holmberg SR, Kar B, Treichel AJ, Ichino N, Liu W, Yang J, Ding Y, Deng Y, Lacey JM, Laxen WJ, Loken PR, Oglesbee D, Farber SA, Clark KJ, Xu X, Ekker SC. Sabharwal A, et al. Among authors: loken pr. Elife. 2022 Nov 21;11:e65488. doi: 10.7554/eLife.65488. Elife. 2022. PMID: 36408801 Free PMC article.
Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria.
McGregor TL, Hunt KA, Yee E, Mason D, Nioi P, Ticau S, Pelosi M, Loken PR, Finer S, Lawlor DA, Fauman EB, Huang QQ, Griffiths CJ, MacArthur DG, Trembath RC, Oglesbee D, Lieske JC, Erbe DV, Wright J, van Heel DA. McGregor TL, et al. Among authors: loken pr. Elife. 2020 Mar 24;9:e54363. doi: 10.7554/eLife.54363. Elife. 2020. PMID: 32207686 Free PMC article.