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676 results

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Page 1
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. Among authors: weiss mj. 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.
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR. Newby GA, et al. Among authors: weiss mj. Nature. 2021 Jul;595(7866):295-302. doi: 10.1038/s41586-021-03609-w. Epub 2021 Jun 2. Nature. 2021. PMID: 34079130 Free PMC article.
Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.
Qin K, Huang P, Feng R, Keller CA, Peslak SA, Khandros E, Saari MS, Lan X, Mayuranathan T, Doerfler PA, Abdulmalik O, Giardine B, Chou ST, Shi J, Hardison RC, Weiss MJ, Blobel GA. Qin K, et al. Among authors: weiss mj. Nat Genet. 2022 Jun;54(6):874-884. doi: 10.1038/s41588-022-01076-1. Epub 2022 May 26. Nat Genet. 2022. PMID: 35618846 Free PMC article.
Activation of γ-globin expression by hypoxia-inducible factor 1α.
Feng R, Mayuranathan T, Huang P, Doerfler PA, Li Y, Yao Y, Zhang J, Palmer LE, Mayberry K, Christakopoulos GE, Xu P, Li C, Cheng Y, Blobel GA, Simon MC, Weiss MJ. Feng R, et al. Among authors: weiss mj. Nature. 2022 Oct;610(7933):783-790. doi: 10.1038/s41586-022-05312-w. Epub 2022 Oct 12. Nature. 2022. PMID: 36224385 Free PMC article.
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. Among authors: weiss mj. 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 of HBG1 and HBG2 to induce fetal hemoglobin.
Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ. Métais JY, et al. Among authors: weiss mj. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing.
Leibowitz ML, Papathanasiou S, Doerfler PA, Blaine LJ, Sun L, Yao Y, Zhang CZ, Weiss MJ, Pellman D. Leibowitz ML, et al. Among authors: weiss mj. Nat Genet. 2021 Jun;53(6):895-905. doi: 10.1038/s41588-021-00838-7. Epub 2021 Apr 12. Nat Genet. 2021. PMID: 33846636 Free PMC article.
Genetic therapies for the first molecular disease.
Doerfler PA, Sharma A, Porter JS, Zheng Y, Tisdale JF, Weiss MJ. Doerfler PA, et al. Among authors: weiss mj. J Clin Invest. 2021 Apr 15;131(8):e146394. doi: 10.1172/JCI146394. J Clin Invest. 2021. PMID: 33855970 Free PMC article. Review.
676 results