Toward ‘n of one’ gene therapies: Scalable ex vivo adenine base editing via lipid nanoparticles in a sickle cell disease model

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Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1. 1Multiplex base editing enhances fetal hemoglobin production in sickle cell disease erythroid cells2025
2. 2In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers2025 · 2 citations
3. 3In Vivo RNA delivery by targeted lipid nanoparticles enable gene editing in hematopoietic stem cells and T cells2025 · 5 citations
4. 4Lipid nanoparticle-mediated epitope editing in human HSPCs enables resistance to CD45-directed CAR-T cell therapy2025 · 1 citations
5. 5Base editing of β 0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies2025