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26 results

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Page 1
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Wu Y, et al. Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25. Nat Med. 2019. PMID: 30911135 Free PMC article.
CRISPResso2 provides accurate and rapid genome editing sequence analysis.
Clement K, Rees H, Canver MC, Gehrke JM, Farouni R, Hsu JY, Cole MA, Liu DR, Joung JK, Bauer DE, Pinello L. Clement K, et al. Nat Biotechnol. 2019 Mar;37(3):224-226. doi: 10.1038/s41587-019-0032-3. Nat Biotechnol. 2019. PMID: 30809026 Free PMC article. No abstract available.
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells.
Nagpal N, Wang J, Zeng J, Lo E, Moon DH, Luk K, Braun RO, Burroughs LM, Keel SB, Reilly C, Lindsley RC, Wolfe SA, Tai AK, Cahan P, Bauer DE, Fong YW, Agarwal S. Nagpal N, et al. Cell Stem Cell. 2020 Jun 4;26(6):896-909.e8. doi: 10.1016/j.stem.2020.03.016. Epub 2020 Apr 21. Cell Stem Cell. 2020. PMID: 32320679 Free PMC article.
Whole genome sequencing identifies structural variants contributing to hematologic traits in the NHLBI TOPMed program.
Wheeler MM, Stilp AM, Rao S, Halldórsson BV, Beyter D, Wen J, Mihkaylova AV, McHugh CP, Lane J, Jiang MZ, Raffield LM, Jun G, Sedlazeck FJ, Metcalf G, Yao Y, Bis JB, Chami N, de Vries PS, Desai P, Floyd JS, Gao Y, Kammers K, Kim W, Moon JY, Ratan A, Yanek LR, Almasy L, Becker LC, Blangero J, Cho MH, Curran JE, Fornage M, Kaplan RC, Lewis JP, Loos RJF, Mitchell BD, Morrison AC, Preuss M, Psaty BM, Rich SS, Rotter JI, Tang H, Tracy RP, Boerwinkle E, Abecasis GR, Blackwell TW, Smith AV, Johnson AD, Mathias RA, Nickerson DA, Conomos MP, Li Y, Þorsteinsdóttir U, Magnússon MK, Stefansson K, Pankratz ND, Bauer DE, Auer PL, Reiner AP. Wheeler MM, et al. Nat Commun. 2022 Dec 8;13(1):7592. doi: 10.1038/s41467-022-35354-7. Nat Commun. 2022. PMID: 36481753 Free PMC article.
ZNF410 represses fetal globin by singular control of CHD4.
Vinjamur DS, Yao Q, Cole MA, McGuckin C, Ren C, Zeng J, Hossain M, Luk K, Wolfe SA, Pinello L, Bauer DE. Vinjamur DS, et al. Nat Genet. 2021 May;53(5):719-728. doi: 10.1038/s41588-021-00843-w. Epub 2021 Apr 15. Nat Genet. 2021. PMID: 33859416 Free PMC article.
Molecular analysis of the erythroid phenotype of a patient with BCL11A haploinsufficiency.
Wessels MW, Cnossen MH, van Dijk TB, Gillemans N, Schmidt KLJ, van Lom K, Vinjamur DS, Coyne S, Kurita R, Nakamura Y, de Man SA, Pfundt R, Azmani Z, Brouwer RWW, Bauer DE, van den Hout MCGN, van IJcken WFJ, Philipsen S. Wessels MW, et al. Blood Adv. 2021 May 11;5(9):2339-2349. doi: 10.1182/bloodadvances.2020003753. Blood Adv. 2021. PMID: 33938942 Free PMC article.
26 results