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Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Mamcarz E, et al. Among authors: ryu by. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. N Engl J Med. 2019. PMID: 30995372 Free PMC article. Clinical Trial.
Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.
Ozog S, Timberlake ND, Hermann K, Garijo O, Haworth KG, Shi G, Glinkerman CM, Schefter LE, D'Souza S, Simpson E, Sghia-Hughes G, Carillo RR, Boger DL, Kiem HP, Slukvin I, Ryu BY, Sorrentino BP, Adair JE, Snyder SA, Compton AA, Torbett BE. Ozog S, et al. Among authors: ryu by. Blood. 2019 Oct 17;134(16):1298-1311. doi: 10.1182/blood.2019000040. Blood. 2019. PMID: 31416800 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: ryu by. 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. 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. 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.
126 results