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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. Among authors: li a. 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.
A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing.
Li A, Lee CM, Hurley AE, Jarrett KE, De Giorgi M, Lu W, Balderrama KS, Doerfler AM, Deshmukh H, Ray A, Bao G, Lagor WR. Li A, et al. Mol Ther Methods Clin Dev. 2018 Dec 6;12:111-122. doi: 10.1016/j.omtm.2018.11.009. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30619914 Free PMC article.
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.
Li A, Tanner MR, Lee CM, Hurley AE, De Giorgi M, Jarrett KE, Davis TH, Doerfler AM, Bao G, Beeton C, Lagor WR. Li A, et al. Mol Ther. 2020 Jun 3;28(6):1432-1441. doi: 10.1016/j.ymthe.2020.04.017. Epub 2020 Apr 19. Mol Ther. 2020. PMID: 32348718 Free PMC article.
Targeting the Apoa1 locus for liver-directed gene therapy.
De Giorgi M, Li A, Hurley A, Barzi M, Doerfler AM, Cherayil NA, Smith HE, Brown JD, Lin CY, Bissig KD, Bao G, Lagor WR. De Giorgi M, et al. Among authors: li a. Mol Ther Methods Clin Dev. 2021 Apr 24;21:656-669. doi: 10.1016/j.omtm.2021.04.011. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 34141821 Free PMC article.
Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment.
Doerfler AM, Han J, Jarrett KE, Tang L, Jain A, Saltzman A, De Giorgi M, Chuecos M, Hurley AE, Li A, Morand P, Ayala C, Goodlett DR, Malovannaya A, Martin JF, de Aguiar Vallim TQ, Shroyer N, Lagor WR. Doerfler AM, et al. Among authors: li a. Arterioscler Thromb Vasc Biol. 2022 Apr;42(4):381-394. doi: 10.1161/ATVBAHA.122.317320. Epub 2022 Feb 17. Arterioscler Thromb Vasc Biol. 2022. PMID: 35172604 Free PMC article.
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study.
Doerfler AM, Park SH, Assini JM, Youssef A, Saxena L, Yaseen AB, De Giorgi M, Chuecos M, Hurley AE, Li A, Marcovina SM, Bao G, Boffa MB, Koschinsky ML, Lagor WR. Doerfler AM, et al. Among authors: li a. Mol Ther Methods Clin Dev. 2022 Oct 13;27:337-351. doi: 10.1016/j.omtm.2022.10.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36381302 Free PMC article.
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