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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: gamer j. J Clin Invest. 2020 Dec 1;130(12):6677-6687. doi: 10.1172/JCI140189. J Clin Invest. 2020. PMID: 32897878 Free PMC article.
βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells.
Demirci S, Gudmundsdottir B, Li Q, Haro-Mora JJ, Nassehi T, Drysdale C, Yapundich M, Gamer J, Seifuddin F, Tisdale JF, Uchida N. Demirci S, et al. Among authors: gamer j. Mol Ther Methods Clin Dev. 2020 Apr 18;17:912-921. doi: 10.1016/j.omtm.2020.04.013. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32405513 Free PMC article.
Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models.
Uchida N, Li L, Nassehi T, Drysdale CM, Yapundich M, Gamer J, Haro-Mora JJ, Demirci S, Leonard A, Bonifacino AC, Krouse AE, Linde NS, Allen C, Peshwa MV, De Ravin SS, Donahue RE, Malech HL, Tisdale JF. Uchida N, et al. Among authors: gamer j. Cell Rep Med. 2021 Apr 20;2(4):100247. doi: 10.1016/j.xcrm.2021.100247. eCollection 2021 Apr 20. Cell Rep Med. 2021. PMID: 33948577 Free PMC article.
19 results