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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Lattanzi A, Meneghini V, Pavani G, Amor F, Ramadier S, Felix T, Antoniani C, Masson C, Alibeu O, Lee C, Porteus MH, Bao G, Amendola M, Mavilio F, Miccio A. Lattanzi A, et al. Among authors: porteus mh. Mol Ther. 2019 Jan 2;27(1):137-150. doi: 10.1016/j.ymthe.2018.10.008. Epub 2018 Oct 17. Mol Ther. 2019. PMID: 30424953 Free PMC article.
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
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.
183 results