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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: ryu b. Sci Transl Med. 2021 Apr 28;13(591):eabb0411. doi: 10.1126/scitranslmed.abb0411. Sci Transl Med. 2021. PMID: 33910976
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.
Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media.
Bauler M, Roberts JK, Wu CC, Fan B, Ferrara F, Yip BH, Diao S, Kim YI, Moore J, Zhou S, Wielgosz MM, Ryu B, Throm RE. Bauler M, et al. Among authors: ryu b. Mol Ther Methods Clin Dev. 2019 Nov 26;17:58-68. doi: 10.1016/j.omtm.2019.11.011. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2019. PMID: 31890741 Free PMC article.
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: ryu b. 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.
702 results