Associate Professor Naoya Uchida of The Institute of Medical Science, The University of Tokyo in Japan and co-researchers at National Heart Lung and Blood Institutes (NHLBI) / National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) and St. Jude Children's Research Hospital (SJCRH) in the U.S. demonstrate that co-expression of truncated human erythropoietin receptors (thEpoR) enhances erythropoiesis of gene-modified cells, resulting in improvement of the phenotypic effect of a therapeutic vector in erythroid cells in xenograft mouse and autologous non-human primate transplantation models. It was published in "Science Translational Medicine" on April 28, 2021.
Press release
Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and co-expressed truncated erythropoietin receptor
Description
Science Translational Medicine論文 
Published Article
"Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and co-expressed truncated erythropoietin receptor"
Science Translational Medicine Online April 28, 2021 doi:10.1126/scitranslmed.abb0411
Naoya Uchida1,2*, Francesca Ferrara3, Claire M. Drysdale1, Morgan Yapundich1, Jackson Gamer1, Tina Nassehi1, Julia DiNicola1, Yoshitaka Shibata1, Matthew Wielgosz3, Yoon-Sang Kim3, Matthew Bauler4, Robert E. Throm4, Juan J. Haro-Mora1, Selami Demirci1, Aylin C. Bonifacino5, Allen E. Krouse5, N. Seth Linde5, Robert E. Donahue1, Byoung Ryu3,6, John F. Tisdale1
*Correspondence: NU
Affiliations:
1) Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI) / National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, Maryland (MD) 20892, USA
2) Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan
3) Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, Tennessee (TN) 38105, USA
4) Vector Development and Production Laboratory, SJCRH 38105, Memphis, TN, USA
5) Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA
6) Umoja Biopharma, 1920 Terry Ave, Seattle, Washington (WA) 98101, USA
*Correspondence: NU
Affiliations:
1) Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI) / National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, Maryland (MD) 20892, USA
2) Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan
3) Department of Hematology, St. Jude Children's Research Hospital (SJCRH), Memphis, Tennessee (TN) 38105, USA
4) Vector Development and Production Laboratory, SJCRH 38105, Memphis, TN, USA
5) Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA
6) Umoja Biopharma, 1920 Terry Ave, Seattle, Washington (WA) 98101, USA