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Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease.
Uchida N, Drysdale CM, Nassehi T, Gamer J, Yapundich M, DiNicola J, Shibata Y, Hinds M, Gudmundsdottir B, Haro-Mora JJ, Demirci S, Tisdale JF. Uchida N, et al. Among authors: gamer j. Mol Ther Methods Clin Dev. 2021 Mar 3;21:121-132. doi: 10.1016/j.omtm.2021.02.022. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33816645 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: 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.
Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor.
Uchida N, Ferrara F, Drysdale CM, Yapundich M, Gamer J, Nassehi T, DiNicola J, Shibata Y, Wielgosz M, Kim YS, Bauler M, Throm RE, Haro-Mora JJ, Demirci S, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ryu B, Tisdale JF. Uchida N, et al. Among authors: gamer j. Sci Transl Med. 2021 Apr 28;13(591):eabb0411. doi: 10.1126/scitranslmed.abb0411. Sci Transl Med. 2021. PMID: 33910976
13 results