Associate Professor Naoya Uchida of The Institute of Medical Science, The University of Tokyo in Japan and co-researchers at the National Heart Lung and Blood Institutes (NHLBI) / the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institutes of Health (NIH) in the USA comprehensively discussed hematopoietic stem cell gene-addition and gene-editing therapies for sickle cell disease (SCD) and clarified the points necessary to develop a new hematopoietic stem cell therapy with genetic modification. It was published in "Cell Stem Cell" on February 4, 2021.
Press release
Hematopoietic stem cell-targeted gene-addition and gene-editing strategies for β-hemoglobinopathies
Description
Published Article
"Hematopoietic stem cell-targeted gene-addition and gene-editing strategies for β-hemoglobinopathies"
Cell Stem Cell February 4，2021 doi:10.1016/j.stem.2021.01.001
Claire M. Drysdale1†, Tina Nassehi1†, Jackson Gamer1†, Morgan Yapundich1†, John F. Tisdale1*, Naoya Uchida1,2*
†Equal contributions: CMD, TN, JG, and MY. *Correspondence: JT, 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
†Equal contributions: CMD, TN, JG, and MY. *Correspondence: JT, 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
