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2017 2
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2023 8
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21 results

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Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS. Mayuranathan T, et al. Nat Genet. 2023 Jul;55(7):1210-1220. doi: 10.1038/s41588-023-01434-7. Epub 2023 Jul 3. Nat Genet. 2023. PMID: 37400614 Free PMC article.
Therapeutic Gene Editing Safety and Specificity.
Lux CT, Scharenberg AM. Lux CT, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):787-795. doi: 10.1016/j.hoc.2017.05.002. Epub 2017 Jun 29. Hematol Oncol Clin North Am. 2017. PMID: 28895847 Free PMC article. Review.
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Everette KA, Newby GA, Levine RM, Mayberry K, Jang Y, Mayuranathan T, Nimmagadda N, Dempsey E, Li Y, Bhoopalan SV, Liu X, Davis JR, Nelson AT, Chen PJ, Sousa AA, Cheng Y, Tisdale JF, Weiss MJ, Yen JS, Liu DR. Everette KA, et al. Nat Biomed Eng. 2023 May;7(5):616-628. doi: 10.1038/s41551-023-01026-0. Epub 2023 Apr 17. Nat Biomed Eng. 2023. PMID: 37069266 Free PMC article.
Genome editing in large animal models.
Maynard LH, Humbert O, Peterson CW, Kiem HP. Maynard LH, et al. Mol Ther. 2021 Nov 3;29(11):3140-3152. doi: 10.1016/j.ymthe.2021.09.026. Epub 2021 Oct 1. Mol Ther. 2021. PMID: 34601132 Free PMC article. Review.
CD90-targeted lentiviral vectors for HSC gene therapy.
Berckmueller K, Thomas J, Taha EA, Choo S, Madhu R, Kanestrom G, Rupert PB, Strong R, Kiem HP, Radtke S. Berckmueller K, et al. Mol Ther. 2023 Oct 4;31(10):2901-2913. doi: 10.1016/j.ymthe.2023.08.003. Epub 2023 Aug 7. Mol Ther. 2023. PMID: 37550965
21 results