A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

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5. CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation
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8. Treating Sickle Cell Disease: Gene Therapy Approaches
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9. Current and future gene therapies for hemoglobinopathies
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10. Effective therapies for sickle cell disease: are we there yet?
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11. Development of curative therapies for sickle cell disease
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12. Functional categorization of gene regulatory variants that cause Mendelian conditions
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13. Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy
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14. Gene editing of stem cells for kidney disease modelling and therapeutic intervention
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15. Sickle cell disease: tipping the balance of genomic research to catalyse discoveries in Africa
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16. The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
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17. CRISPR/Cas9 mediated knock-out of VPREB1 gene induces a cytotoxic effect in myeloma cells
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18. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
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19. Red Blood Cells as Therapeutic Target to Treat Sickle Cell Disease
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20. Efficient Delivery and Nuclear Uptake Is Not Sufficient to Detect Gene Editing in CD34+ Cells Directed by a Ribonucleoprotein Complex
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21. Gene Therapy as the New Frontier for Sickle Cell Disease
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22. A Practical Guide to Genome Editing Using Targeted Nuclease Technologies
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24. NRF2 mediates γ-globin gene regulation through epigenetic modifications in a β-YAC transgenic mouse model
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25. The Cas9 Hammer—and Sickle: A Challenge for Genome Editors
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26. Induction of Fetal Hemoglobin by Introducing Natural Hereditary Persistence of Fetal Hemoglobin Mutations in the γ-Globin Gene Promoters for Genome Editing Therapies for β-Thalassemia
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27. FLT1 and other candidate fetal haemoglobin modifying loci in sickle cell disease in African ancestries
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28. A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
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29. Engineering Globin Gene Expression
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31. Innovative Therapies for Hemoglobin Disorders
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32. Genome Editing for Sickle Cell Disease: A Little BCL11A Goes a Long Way
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33. Using Clustered Regularly Interspaced Short Palindromic Repeats gene editing to induce permanent expression of fetal hemoglobin in β-thalassemia and sickle cell disease: A comparative meta-analysis
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34. Advances in Gene Therapy for Sickle Cell Disease: From Preclinical Innovations to Clinical Implementation and Access Challenges
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35. Dissecting the epigenetic regulation of the fetal hemoglobin genes to unravel a novel therapeutic approach for β-hemoglobinopathies
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38. β-Hemoglobinopathies: The Test Bench for Genome Editing-Based Therapeutic Strategies
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40. Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
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42. CRISPR Editing in Biological and Biomedical Investigation
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43. Genetic Therapies for Sickle Cell Disease
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44. Transcriptome Analyses of β-Thalassemia −28(A>G) Mutation Using Isogenic Cell Models Generated by CRISPR/Cas9 and Asymmetric Single-Stranded Oligodeoxynucleotides (assODNs)
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45. In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery
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48. THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES
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