An Erythroid-Specific Lentiviral Vector Improves Anemia and Iron Metabolism in a New Model of Xlsa
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An Erythroid-Specific Lentiviral Vector Improves Anemia and Iron Metabolism in a New Model of Xlsa
Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia
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Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia
In Utero mRNA Lipid Nanoparticle Delivery in a Murine Model of Fanconi Anemia
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In Utero mRNA Lipid Nanoparticle Delivery in a Murine Model of Fanconi Anemia
In Vivo Hematopoietic Stem Cell Engineering Restores the Function of NADPH Enzyme Complex in X-Linked Chronic Granulomatous Disease Model Mice
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In Vivo Hematopoietic Stem Cell Engineering Restores the Function of NADPH Enzyme Complex in X-Linked Chronic Granulomatous Disease Model Mice
Selection for an HIV-Resistant Immune System By Base-Edited CD45 CAR-T Cell Therapy
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Selection for an HIV-Resistant Immune System By Base-Edited CD45 CAR-T Cell Therapy