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Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.
Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Chandler RJ, et al. Among authors: schneller jl. Hepatology. 2021 Jun;73(6):2223-2237. doi: 10.1002/hep.31570. Epub 2021 May 21. Hepatology. 2021. PMID: 32976669 Free PMC article.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV. An D, et al. Among authors: schneller jl. Cell Rep. 2017 Dec 19;21(12):3548-3558. doi: 10.1016/j.celrep.2017.11.081. Cell Rep. 2017. PMID: 29262333 Free PMC article.
In vivo genome editing at the albumin locus to treat methylmalonic acidemia.
Schneller JL, Lee CM, Venturoni LE, Chandler RJ, Li A, Myung S, Cradick TJ, Hurley AE, Lagor WR, Bao G, Venditti CP. Schneller JL, et al. Mol Ther Methods Clin Dev. 2021 Nov 11;23:619-632. doi: 10.1016/j.omtm.2021.11.004. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34901307 Free PMC article.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV. An D, et al. Among authors: schneller jl. Cell Rep. 2018 Aug 28;24(9):2520. doi: 10.1016/j.celrep.2018.08.049. Cell Rep. 2018. PMID: 30157442 Free article. No abstract available.