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The NIH Somatic Cell Genome Editing program.
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. Saha K, et al. Among authors: lagor wr. Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7. Nature. 2021. PMID: 33828315 Free PMC article. Review.
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. Among authors: lagor wr. 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.
Recent advances in genome editing for cardiovascular disease.
Doerfler AM, Walkey CJ, Lagor WR. Doerfler AM, et al. Among authors: lagor wr. Curr Opin Cardiol. 2020 May;35(3):242-248. doi: 10.1097/HCO.0000000000000723. Curr Opin Cardiol. 2020. PMID: 32073405 Free PMC article. Review.
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. Among authors: lagor wr. 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: lagor wr. 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.
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: lagor wr. 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.
66 results