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Therapeutic in vivo delivery of gene editing agents.
Raguram A, Banskota S, Liu DR. Raguram A, et al. Cell. 2022 Jul 21;185(15):2806-2827. doi: 10.1016/j.cell.2022.03.045. Epub 2022 Jul 6. Cell. 2022. PMID: 35798006 Free PMC article. Review.
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Koblan LW, et al. Nature. 2021 Jan;589(7843):608-614. doi: 10.1038/s41586-020-03086-7. Epub 2021 Jan 6. Nature. 2021. PMID: 33408413 Free PMC article.
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.
Efficient prime editing in mouse brain, liver and heart with dual AAVs.
Davis JR, Banskota S, Levy JM, Newby GA, Wang X, Anzalone AV, Nelson AT, Chen PJ, Hennes AD, An M, Roh H, Randolph PB, Musunuru K, Liu DR. Davis JR, et al. Nat Biotechnol. 2024 Feb;42(2):253-264. doi: 10.1038/s41587-023-01758-z. Epub 2023 May 4. Nat Biotechnol. 2024. PMID: 37142705 Free PMC article.
Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo.
Kulhankova K, Traore S, Cheng X, Benk-Fortin H, Hallée S, Harvey M, Roberge J, Couture F, Kohli S, Gross TJ, Meyerholz DK, Rettig GR, Thommandru B, Kurgan G, Wohlford-Lenane C, Hartigan-O'Connor DJ, Yates BP, Newby GA, Liu DR, Tarantal AF, Guay D, McCray PB Jr. Kulhankova K, et al. Nat Commun. 2023 Dec 5;14(1):8051. doi: 10.1038/s41467-023-43904-w. Nat Commun. 2023. PMID: 38052872 Free PMC article.
15 results