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Page 1
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. 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.
Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier.
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. Huang Q, et al. PLoS Biol. 2023 Jul 19;21(7):e3002112. doi: 10.1371/journal.pbio.3002112. eCollection 2023 Jul. PLoS Biol. 2023. PMID: 37467291 Free PMC article.
Promoting validation and cross-phylogenetic integration in model organism research.
Cheng KC, Burdine RD, Dickinson ME, Ekker SC, Lin AY, Lloyd KCK, Lutz CM, MacRae CA, Morrison JH, O'Connor DH, Postlethwait JH, Rogers CD, Sanchez S, Simpson JH, Talbot WS, Wallace DC, Weimer JM, Bellen HJ. Cheng KC, et al. Dis Model Mech. 2022 Sep 1;15(9):dmm049600. doi: 10.1242/dmm.049600. Epub 2022 Sep 20. Dis Model Mech. 2022. PMID: 36125045 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. 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. 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.
18 results