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Cytotherapy|September 19, 2022
Editing human hematopoietic stem cells: advances and challengesSenthil Velan Bhoopalan, Jonathan S Yen, Rachel M Levine, et al.
Science Translational Medicine|August 13, 2025
Engraftment and persistence of <i>HBB</i> base-edited hematopoietic stem cells in nonhuman primatesStefan Radtke, Emily Fields, Kyle Swing, et al.
Blood Advances|February 28, 2025
BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into miceYoonjeong Jang, Ruopeng Feng, Lance E Palmer, et al.
Molecular Therapy. Nucleic Acids|July 6, 2026
Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promotersAnne Chalumeau, Panagiotis Antoniou, Maria Bou Dames, et al.
JCI Insight|November 22, 2022
An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activitySenthil Velan Bhoopalan, Jonathan S Yen, Thiyagaraj Mayuranathan, et al.
Biorxiv : the Preprint Server for Biology|March 3, 2023
Multiplex Base Editing to Protect from CD33-Directed Therapy: Implications for Immune and Gene TherapyFlorence Borot, Olivier Humbert, Gregory A Newby, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2024
Preclinical development of lentiviral vector gene therapy for Diamond-Blackfan anemia syndromeSenthil Velan Bhoopalan, Thiyagaraj Mayuranathan, Nana Liu, et al.
Nature Communications|May 27, 2025
Multiplex base editing to protect from CD33 directed drugs for immune and gene therapyFlorence Borot, Olivier Humbert, Jeffrey T Ehmsen, et al.
Nature Biomedical Engineering|April 17, 2023
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in miceKelcee A Everette, Gregory A Newby, Rachel M Levine, et al.
Nature Nanotechnology|May 23, 2024
Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cellsXizhen Lian, Sumanta Chatterjee, Yehui Sun, et al.
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