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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: shibata y. Sci Transl Med. 2021 Apr 28;13(591):eabb0411. doi: 10.1126/scitranslmed.abb0411. Sci Transl Med. 2021. PMID: 33910976
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: shibata y. 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.