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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.
A Survey of Validation Strategies for CRISPR-Cas9 Editing.
Sentmanat MF, Peters ST, Florian CP, Connelly JP, Pruett-Miller SM. Sentmanat MF, et al. Sci Rep. 2018 Jan 17;8(1):888. doi: 10.1038/s41598-018-19441-8. Sci Rep. 2018. PMID: 29343825 Free PMC article.
The COPII cargo adapter SEC24C is essential for neuronal homeostasis.
Wang B, Joo JH, Mount R, Teubner BJW, Krenzer A, Ward AL, Ichhaporia VP, Adams EJ, Khoriaty R, Peters ST, Pruett-Miller SM, Zakharenko SS, Ginsburg D, Kundu M. Wang B, et al. J Clin Invest. 2018 Aug 1;128(8):3319-3332. doi: 10.1172/JCI98194. Epub 2018 Jun 25. J Clin Invest. 2018. PMID: 29939162 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. J Clin Invest. 2020 Dec 1;130(12):6677-6687. doi: 10.1172/JCI140189. J Clin Invest. 2020. PMID: 32897878 Free PMC article.
136 results