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Publications | Genome Editing Testing Center

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Huang Q, Chen AT, Chan KY, Sorensen H, Barry AJ, Azari B, Zheng Q, Beddow T, Zhao B, Tobey IG, Moncada-Reid C, Eid FE, Walkey CJ, Ljungberg MC, Lagor WR, Heaney JD, Chan YA, Deverman BE. Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier. PLoS Biol. 2023 Jul 19;21(7):e3002112. doi: 10.1371/journal.pbio.3002112. PMID: 37467291.

Lorincz R, Alvarez AB, Walkey CJ, Mendonça SA, Lu ZH, Martinez AE, Ljungberg C, Heaney JD, Lagor WR, Curiel DT. In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector. Biomed Pharmacother. 2023 Feb;158:114189. doi: 10.1016/j.biopha.2022.114189. Epub 2022 Dec 30. PMID: 36587560.

Liang SQ, Walkey CJ, Martinez AE, Su Q, Dickinson ME, Wang D, Lagor WR, Heaney JD, Gao G, Xue W. AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway. Mol Ther. 2022 Jan 5;30(1):238-243. doi: 10.1016/j.ymthe.2021.10.023. Epub 2021 Oct 23. PMID: 34695545; PMCID: PMC8753568.

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. LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study. Mol Ther Methods Clin Dev. 2022 Oct 13;27:337-351. doi: 10.1016/j.omtm.2022.10.009. PMID: 36381302; PMCID: PMC9630778.

Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB Jr, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J; SCGE Consortium. The NIH Somatic Cell Genome Editing program. Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7. PMID: 33828315; PMCID: PMC8026397.

Li A, Tanner MR, Lee CM, Hurley AE, De Giorgi M, Jarrett KE, Davis TH, Doerfler AM, Bao G, Beeton C, Lagor WR. AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9. Mol Ther. 2020 Jun 3;28(6):1432-1441. doi: 10.1016/j.ymthe.2020.04.017. Epub 2020 Apr 19. PMID: 32348718; PMCID: PMC7264438.

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. A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing. Mol Ther Methods Clin Dev. 2018 Dec 6;12:111-122. doi: 10.1016/j.omtm.2018.11.009. PMID: 30619914; PMCID: PMC6313841.