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Page 1
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: tisdale jf. 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: tisdale jf. 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. Among authors: tisdale jf. J Clin Invest. 2020 Dec 1;130(12):6677-6687. doi: 10.1172/JCI140189. J Clin Invest. 2020. PMID: 32897878 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: tisdale jf. J Clin Invest. 2021 Apr 15;131(8):e146394. doi: 10.1172/JCI146394. J Clin Invest. 2021. PMID: 33855970 Free PMC article. Review.
Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease.
Leonard A, Sharma A, Uchida N, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Leonard A, et al. Among authors: tisdale jf. Blood Adv. 2021 May 11;5(9):2403-2411. doi: 10.1182/bloodadvances.2021004232. Blood Adv. 2021. PMID: 33956057 Free PMC article. Clinical Trial.
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: tisdale jf. 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.
Optimizing haematopoietic stem and progenitor cell apheresis collection from plerixafor-mobilized patients with sickle cell disease.
Sharma A, Leonard A, West K, Gossett JM, Uchida N, Panch S, Stroncek D, Poston L, Akel S, Hankins JS, Fitzhugh C, Hsieh MM, Kang G, Tisdale JF, Weiss MJ, Zheng Y. Sharma A, et al. Among authors: tisdale jf. Br J Haematol. 2022 Aug;198(4):740-744. doi: 10.1111/bjh.18311. Epub 2022 Jun 23. Br J Haematol. 2022. PMID: 35737751
Forced enhancer-promoter rewiring to alter gene expression in animal models.
Peslak SA, Demirci S, Chandra V, Ryu B, Bhardwaj SK, Jiang J, Rupon JW, Throm RE, Uchida N, Leonard A, Essawi K, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ferrara F, Wielgosz M, Abdulmalik O, Hamagami N, Germino-Watnick P, Le A, Chu R, Hinds M, Weiss MJ, Tong W, Tisdale JF, Blobel GA. Peslak SA, et al. Among authors: tisdale jf. Mol Ther Nucleic Acids. 2023 Jan 31;31:452-465. doi: 10.1016/j.omtn.2023.01.016. eCollection 2023 Mar 14. Mol Ther Nucleic Acids. 2023. PMID: 36852088 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. Among authors: tisdale jf. 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.
Gene therapy for sickle cell disease.
Leonard A, Tisdale JF. Leonard A, et al. Among authors: tisdale jf. Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):542-547. doi: 10.1182/hematology.2023000487. Hematology Am Soc Hematol Educ Program. 2023. PMID: 38066927
228 results