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
解説
プレスリリース
論文情報
"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)
Science Translational Medicine論文
Cell Reports Medicine論文
発表解説論文(English Pape1:Science Translational Medicine)
発表解説論文(English Page2:Cell Reports Medicine)