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Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I.
Gomez-Ospina N, Scharenberg SG, Mostrel N, Bak RO, Mantri S, Quadros RM, Gurumurthy CB, Lee C, Bao G, Suarez CJ, Khan S, Sawamoto K, Tomatsu S, Raj N, Attardi LD, Aurelian L, Porteus MH. Gomez-Ospina N, et al. Among authors: porteus mh. Nat Commun. 2019 Sep 6;10(1):4045. doi: 10.1038/s41467-019-11962-8. Nat Commun. 2019. PMID: 31492863 Free PMC article.
Receptor-mediated delivery of engineered nucleases for genome modification.
Chen Z, Jaafar L, Agyekum DG, Xiao H, Wade MF, Kumaran RI, Spector DL, Bao G, Porteus MH, Dynan WS, Meiler SE. Chen Z, et al. Among authors: porteus mh. Nucleic Acids Res. 2013 Oct;41(19):e182. doi: 10.1093/nar/gkt710. Epub 2013 Aug 16. Nucleic Acids Res. 2013. PMID: 23956220 Free PMC article.
SAPTA: a new design tool for improving TALE nuclease activity.
Lin Y, Fine EJ, Zheng Z, Antico CJ, Voit RA, Porteus MH, Cradick TJ, Bao G. Lin Y, et al. Among authors: porteus mh. Nucleic Acids Res. 2014 Apr;42(6):e47. doi: 10.1093/nar/gkt1363. Epub 2014 Jan 16. Nucleic Acids Res. 2014. PMID: 24442582 Free PMC article.
Quantifying on- and off-target genome editing.
Hendel A, Fine EJ, Bao G, Porteus MH. Hendel A, et al. Among authors: porteus mh. Trends Biotechnol. 2015 Feb;33(2):132-40. doi: 10.1016/j.tibtech.2014.12.001. Epub 2015 Jan 13. Trends Biotechnol. 2015. PMID: 25595557 Free PMC article. Review.
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.
Hendel A, Bak RO, Clark JT, Kennedy AB, Ryan DE, Roy S, Steinfeld I, Lunstad BD, Kaiser RJ, Wilkens AB, Bacchetta R, Tsalenko A, Dellinger D, Bruhn L, Porteus MH. Hendel A, et al. Among authors: porteus mh. Nat Biotechnol. 2015 Sep;33(9):985-989. doi: 10.1038/nbt.3290. Epub 2015 Jun 29. Nat Biotechnol. 2015. PMID: 26121415 Free PMC article.
MLL leukemia induction by genome editing of human CD34+ hematopoietic cells.
Buechele C, Breese EH, Schneidawind D, Lin CH, Jeong J, Duque-Afonso J, Wong SH, Smith KS, Negrin RS, Porteus M, Cleary ML. Buechele C, et al. Blood. 2015 Oct 1;126(14):1683-94. doi: 10.1182/blood-2015-05-646398. Epub 2015 Aug 26. Blood. 2015. PMID: 26311362 Free PMC article.
Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis.
Gomez-Ospina N, Potter CJ, Xiao R, Manickam K, Kim MS, Kim KH, Shneider BL, Picarsic JL, Jacobson TA, Zhang J, He W, Liu P, Knisely AS, Finegold MJ, Muzny DM, Boerwinkle E, Lupski JR, Plon SE, Gibbs RA, Eng CM, Yang Y, Washington GC, Porteus MH, Berquist WE, Kambham N, Singh RJ, Xia F, Enns GM, Moore DD. Gomez-Ospina N, et al. Among authors: porteus mh. Nat Commun. 2016 Feb 18;7:10713. doi: 10.1038/ncomms10713. Nat Commun. 2016. PMID: 26888176 Free PMC article.
190 results