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Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.
Bonafont J, Mencía A, Chacón-Solano E, Srifa W, Vaidyanathan S, Romano R, Garcia M, Hervás-Salcedo R, Ugalde L, Duarte B, Porteus MH, Del Rio M, Larcher F, Murillas R. Bonafont J, et al. Among authors: porteus mh. Mol Ther. 2021 Jun 2;29(6):2008-2018. doi: 10.1016/j.ymthe.2021.02.019. Epub 2021 Feb 18. Mol Ther. 2021. PMID: 33609734 Free PMC article.
Knock-in editing: it functionally corrects!
Porteus MH. Porteus MH. Blood. 2016 May 26;127(21):2507-9. doi: 10.1182/blood-2016-03-703181. Blood. 2016. PMID: 27231391 Free PMC article.
Gene Editing on Center Stage.
Bak RO, Gomez-Ospina N, Porteus MH. Bak RO, et al. Among authors: porteus mh. Trends Genet. 2018 Aug;34(8):600-611. doi: 10.1016/j.tig.2018.05.004. Epub 2018 Jun 13. Trends Genet. 2018. PMID: 29908711 Review.
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH. Cromer MK, et al. Among authors: porteus mh. Mol Ther. 2018 Oct 3;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002. Epub 2018 Jul 11. Mol Ther. 2018. PMID: 30005866 Free PMC article.
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification.
Vaidyanathan S, Azizian KT, Haque AKMA, Henderson JM, Hendel A, Shore S, Antony JS, Hogrefe RI, Kormann MSD, Porteus MH, McCaffrey AP. Vaidyanathan S, et al. Among authors: porteus mh. Mol Ther Nucleic Acids. 2018 Sep 7;12:530-542. doi: 10.1016/j.omtn.2018.06.010. Epub 2018 Jun 30. Mol Ther Nucleic Acids. 2018. PMID: 30195789 Free PMC article.
181 results