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), and National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH) as well as MaxCyte, Inc., in the U.S. developed a viral vector-free non-footprint gene correction method for the SCD mutation at therapeutic levels in DNA (~30%) and proteins (~80%) and demonstrated engraftment of gene-edited CD34+ cells post-transplant in xenograft mice and non-human primates. It was published in "Cell Reports Medicine" on April 20, 2021.
Press release
Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease (SCD) locus in xenograft mouse and non-human primate models
Description
Published Article
"Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models"
Cell Reports Medicine Online April 20, 2021 doi:10.1016/j.xcrm.2021.100247
Naoya Uchida1,2*, Linhong Li3, Tina Nassehi1, Claire M. Drysdale1, Morgan Yapundich1, Jackson Gamer1, Juan J. Haro-Mora1, Selami Demirci1, Alexis Leonard1, Aylin C. Bonifacino4, Allen E. Krouse4, N. Seth Linde4, Cornell Allen3, Madhusudan V. Peshwa3, Suk See De Ravin5, Robert E. Donahue1, Harry L Malech5, and 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), 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, Japan
3) MaxCyte, Inc., Gaithersburg, MD, USA
4) Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA
5) Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, 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), 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, Japan
3) MaxCyte, Inc., Gaithersburg, MD, USA
4) Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA
5) Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, USA
