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Page 1
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. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.
Demirci S, Zeng J, Wu Y, Uchida N, Shen AH, Pellin D, Gamer J, Yapundich M, Drysdale C, Bonanno J, Bonifacino AC, Krouse AE, Linde NS, Engels T, Donahue RE, Haro-Mora JJ, Leonard A, Nassehi T, Luk K, Porter SN, Lazzarotto CR, Tsai SQ, Weiss MJ, Pruett-Miller SM, Wolfe SA, Bauer DE, Tisdale JF. Demirci S, et al. J Clin Invest. 2020 Dec 1;130(12):6677-6687. doi: 10.1172/JCI140189. J Clin Invest. 2020. PMID: 32897878 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. 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.
Degradation of Janus kinases in CRLF2-rearranged acute lymphoblastic leukemia.
Chang Y, Min J, Jarusiewicz JA, Actis M, Yu-Chen Bradford S, Mayasundari A, Yang L, Chepyala D, Alcock LJ, Roberts KG, Nithianantham S, Maxwell D, Rowland L, Larsen R, Seth A, Goto H, Imamura T, Akahane K, Hansen BS, Pruett-Miller SM, Paietta EM, Litzow MR, Qu C, Yang JJ, Fischer M, Rankovic Z, Mullighan CG. Chang Y, et al. Blood. 2021 Dec 9;138(23):2313-2326. doi: 10.1182/blood.2020006846. Blood. 2021. PMID: 34110416 Free PMC article.
Amino acid stress response genes promote L-asparaginase resistance in pediatric acute lymphoblastic leukemia.
Ferguson DC, McCorkle JR, Barnett KR, Bonten EJ, Bergeron BP, Bhattarai KR, Yang W, Smith C, Hansen BS, Bajpai R, Dong Q, Autry RJ, Gocho Y, Diedrich JD, Crews KR, Pruett-Miller SM, Roberts KG, Stock W, Mullighan CG, Inaba H, Jeha S, Pui CH, Yang JJ, Relling MV, Evans WE, Savic D. Ferguson DC, et al. Blood Adv. 2022 Jun 14;6(11):3386-3397. doi: 10.1182/bloodadvances.2022006965. Blood Adv. 2022. PMID: 35671062 Free PMC article.
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.
Foxp3 enhancers synergize to maximize regulatory T cell suppressive capacity.
Zong X, Hao X, Xu B, Crawford JC, Wright S, Li J, Zhang Y, Bai L, He M, Jiang M, Fan Y, Connelly JP, Pruett-Miller SM, Berns H, Janke L, Li C, Feng Y. Zong X, et al. J Exp Med. 2021 Aug 2;218(8):e20202415. doi: 10.1084/jem.20202415. Epub 2021 Jun 4. J Exp Med. 2021. PMID: 34086055 Free PMC article.
138 results