鎌状赤血球貧血症に対する造血幹細胞遺伝子付加・遺伝子修復治療の開発研究

"Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and co-expressed truncated erythropoietin receptor" / "Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models"

Science Translational Medicine/Cell Reports Medicine オンライン版 2021年4月28日/2021年4月20日 doi:10.1126/scitranslmed.abb0411/10.1016/j.xcrm.2021.100247

雑誌名：「Science Translational Medicine」
論文タイトル：Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and co-expressed truncated erythropoietin receptor
著者：Naoya Uchida*, Francesca Ferrara, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Tina Nassehi, Julia DiNicola, Yoshitaka Shibata, Matthew Wielgosz, Yoon-Sang Kim, Matthew Bauler, Robert E. Throm, Juan J. Haro-Mora, Selami Demirci, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Robert E. Donahue, Byoung Ryu, John F. Tisdale (*Correspondence: NU)


雑誌名：「Cell Reports Medicine」
論文タイトル：Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
著者：Naoya Uchida*, Linhong Li, Tina Nassehi, Claire M. Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro-Mora, Selami Demirci, Alexis Leonard, Aylin C. Bonifacino, Allen E. Krouse, N. Seth Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L Malech, and John F. Tisdale (*Correspondence: NU)