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Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome.
Pille M, Avila JM, Park SH, Le CQ, Xue H, Haerynck F, Saxena L, Lee C, Shpall EJ, Bao G, Vandekerckhove B, Davis BR. Pille M, et al. Among authors: saxena l. Mol Ther Methods Clin Dev. 2024 Feb 6;32(1):101208. doi: 10.1016/j.omtm.2024.101208. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38414825 Free PMC article.
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.
De Giorgi M, Park SH, Castoreno A, Cao M, Hurley A, Saxena L, Chuecos MA, Walkey CJ, Doerfler AM, Furgurson MN, Ljungberg MC, Patel KR, Hyde S, Chickering T, Lefebvre S, Wassarman K, Miller P, Qin J, Schlegel MK, Zlatev I, Li RG, Kim J, Martin JF, Bissig KD, Jadhav V, Bao G, Lagor WR. De Giorgi M, et al. Among authors: saxena l. bioRxiv [Preprint]. 2023 Jul 29:2023.07.26.550728. doi: 10.1101/2023.07.26.550728. bioRxiv. 2023. PMID: 37546995 Free PMC article. Preprint.
High levels of lipoprotein(a) in transgenic mice exacerbate atherosclerosis and promote vulnerable plaque features in a sex-specific manner.
Assini JM, Clark JR, Youssef A, Xing C, Doerfler AM, Park SH, Saxena L, Yaseen AB, Børen J, Gros R, Bao G, Lagor WR, Boffa MB, Koschinsky ML. Assini JM, et al. Among authors: saxena l. Atherosclerosis. 2023 Nov;384:117150. doi: 10.1016/j.atherosclerosis.2023.05.019. Epub 2023 May 30. Atherosclerosis. 2023. PMID: 37290980
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: saxena l. 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.
28 results