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Nature Medicine|March 19, 2021
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cellsM Kyle Cromer, Joab Camarena, Renata M Martin, et al.Blood|February 24, 2021
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cellsSuk See De Ravin, Julie Brault, Ronald J Meis, et al.Blood Advances|December 14, 2023
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiencyMara Pavel-Dinu, Cameron L Gardner, Yusuke Nakauchi, et al.Journal of Immunology (Baltimore, Md. : 1950)|July 9, 2008
CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma modelArvind Chhabra, Lili Yang, Pin Wang, et al.Proceedings of the National Academy of Sciences of the United States of America|October 24, 2018
Isolation and characterization of NY-ESO-1-specific T cell receptors restricted on various MHC moleculesMichael T Bethune, Xiao-Hua Li, Jiaji Yu, et al.Cell Stem Cell|December 17, 2019
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated EpitheliaSriram Vaidyanathan, Ameen A Salahudeen, Zachary M Sellers, et al.Developmental Cell|April 3, 2024
Lineage-tracing hematopoietic stem cell origins in vivo to efficiently make human HLF+ HOXA+ hematopoietic progenitors from pluripotent stem cellsJonas L Fowler, Sherry Li Zheng, Alana Nguyen, et al.Cell|June 23, 2022
Generating human artery and vein cells from pluripotent stem cells highlights the arterial tropism of Nipah and Hendra virusesLay Teng Ang, Alana T Nguyen, Kevin J Liu, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 1, 2025
A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expressionKirby A Wallace, Trevor L Gerstenberg, Craig L Ennis, et al.Nature Medicine|September 29, 2020
Neuronal defects in a human cellular model of 22q11.2 deletion syndromeThemasap A Khan, Omer Revah, Aaron Gordon, et al.Pageof 28