Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

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Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain

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Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

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CRISPR/Cas9 gene editing for curing sickle cell disease

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Transcriptional Silencers: Driving Gene Expression with the Brakes On

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Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

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Rapid and reproducible characterization of sickling during automated deoxygenation in sickle cell disease patients

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CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells

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A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site

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Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: A promising approach for gene therapy of beta thalassemia disease

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Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease

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Evolving AAV-delivered therapeutics towards ultimate cures

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Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

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2021 update on clinical trials in β‐thalassemia

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Gene Therapy for Hemoglobinopathies

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Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin

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TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

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Comparing the two leading erythroid lines BEL-A and HUDEP-2

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CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy

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Therapy Development by Genome Editing of Hematopoietic Stem Cells

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Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

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NEW THERAPEUTIC OPTIONS FOR THE TREATMENT OF SICKLE CELL DISEASE

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CRISPR/Cas9-based gene-editing technology for sickle cell disease

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Revolutionising healing: Gene Editing's breakthrough against sickle cell disease

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CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

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Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin)

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Improved Cas9 activity by specific modifications of the tracrRNA

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Oxygen gradient ektacytometry‐derived biomarkers are associated with vaso‐occlusive crises and correlate with treatment response in sickle cell disease

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A unified model of human hemoglobin switching through single-cell genome editing

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Genome Editing for β-Hemoglobinopathies: Advances and Challenges

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CRISPR/Cas9 increases mitotic gene conversion in human cells

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βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

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Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders

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Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy

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Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies

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Treatment of Erythroid Precursor Cells from β-Thalassemia Patients with Cinchona Alkaloids: Induction of Fetal Hemoglobin Production

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CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

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Beta‐thalassemia and the advent of new interventions beyond transfusion and iron chelation

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Advances in therapeutic application of CRISPR-Cas9

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HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe

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EHA Research Roadmap on Hemoglobinopathies and Thalassemia: An Update

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Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies

Venkatesan, V., Srinivasan, S., Babu, P., & Thangavel, S. (2020) · Molecular and Cellular Biology · doi:10.1128/mcb.00253-20 · Cited by 22

Society for Maternal-Fetal Medicine Special Statement: Beyond the scalpel: in utero fetal gene therapy and curative medicine

Shanahan, M. A., Aagaard, K. M., McCullough, L. B., Chervenak, F. A., & Shamshirsaz, A. A. (2021) · American Journal of Obstetrics and Gynecology · doi:10.1016/j.ajog.2021.09.001 · Cited by 22

CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression

Han, Y., Tan, X., Jin, T., Zhao, S., Li, H., Zhang, W., Kurita, R., Nakamura, Y., Liu, J., Li, D., et al. (2022) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2022.174788 · Cited by 21

Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

Papizan, J. B., Porter, S. N., Sharma, A., & Pruett‐Miller, S. M. (2020) · Journal of Biomedical Research · doi:10.7555/jbr.34.20200096 · Cited by 21

Advances in CRISPR/Cas gene therapy for inborn errors of immunity

Liu, X., Li, G., Liu, Y., Zhou, F., Huang, X., & Li, K. (2023) · Frontiers in Immunology · doi:10.3389/fimmu.2023.1111777 · Cited by 21

Genome-based therapeutic interventions for β-type hemoglobinopathies

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Establishment and characterization of immortalized erythroid progenitor cell lines derived from a common cell source

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A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

Zittersteijn, H. A., Harteveld, C. L., Klaver-Flores, S., Lankester, A. C., Hoeben, R. C., Staal, F. J. T., & Gonçalves, M. A. F. V. (2021) · Frontiers in Genome Editing · doi:10.3389/fgeed.2020.617780 · Cited by 21

In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations

Psatha, N., Paschoudi, K., Παπαδοπούλου, Α., & Yannaki, E. (2022) · Genes · doi:10.3390/genes13122222 · Cited by 24

Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

Brusson, M., Chalumeau, A., Martinucci, P., Romano, O., Félix, T., Poletti, V., Scaramuzza, S., Ramadier, S., Masson, C., Ferrari, G., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.03.012 · Cited by 23

Oxygen gradient ektacytometry–derived biomarkers are associated with acute complications in sickle cell disease

Rab, M. A. E., Kanne, C. K., Boisson, C., Bos, J., van Oirschot, B. A., Houwing, M. E., Renoux, C., Bartels, M., Rijneveld, A. W., Nur, E., et al. (2023) · Blood Advances · doi:10.1182/bloodadvances.2023011013 · Cited by 18

SEC23A rescues SEC23B-deficient congenital dyserythropoietic anemia type II

King, R., Lin, Z., Balbin-Cuesta, G., Myers, G., Friedman, A. D., Zhu, G., McGee, B. M., Saunders, T. L., Kurita, R., Nakamura, Y., et al. (2021) · Science Advances · doi:10.1126/sciadv.abj5293 · Cited by 20

CRISPR/Cas9, a promising approach for the treatment of β-thalassemia: a systematic review

Khiabani, A., Kohansal, M. H., Keshavarzi, A., Shahraki, H., Kooshesh, M., Karimzade, M., & Navashenaq, J. G. (2022) · Molecular Genetics and Genomics · doi:10.1007/s00438-022-01978-z · Cited by 18

CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases

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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

Ramadier, S., Chalumeau, A., Félix, T., Othman, N., Aknoun, S., Casini, A., Maule, G., Masson, C., Cian, A. D., Frati, G., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.08.019 · Cited by 17

Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies

Venkatesan, V., Christopher, A. C., Rhiel, M., Azhagiri, M. K. K., Babu, P., Walavalkar, K., Saravanan, B., Andrieux, G., Rangaraj, S., Srinivasan, S., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.04.024 · Cited by 18

Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

Mattè, A., Cappellini, M. D., Iolascon, A., Federti, E., & Franceschi, L. D. (2019) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2020.1703947 · Cited by 17

Induction of therapeutic levels of HbF in genome‐edited primary β039‐thalassaemia haematopoietic stem and progenitor cells

Mingoia, M., Caria, C. A., Ye, L., Asunis, I., Marongiu, M. F., Manunza, L., Sollaino, M. C., Wang, J., Cabriolu, A., Kurita, R., et al. (2020) · British Journal of Haematology · doi:10.1111/bjh.17167 · Cited by 18

Target-enriched nanopore sequencing and de novo assembly reveals co-occurrences of complex on-target genomic rearrangements induced by CRISPR-Cas9 in human cellsPreprint

Geng, K., Merino, L. G., Wedemann, L., Martens, A., Sobota, M., Sanchez, Y. P., Søndergaard, J. N., White, R. J., & Kutter, C. (2022) · Genome Research · doi:10.1101/gr.276901.122 · Cited by 19

Recent advancements in gene therapy for sickle cell disease and β-thalassemia

Li, L., & Mandal, P. K. (2024) · Frontiers in Hematology · doi:10.3389/frhem.2024.1468952 · Cited by 23

Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples

Modarai, S., Kanda, S., Bloh, K. M., Opdenaker, L. M., & Kmiec, E. B. (2020) · Gene Therapy · doi:10.1038/s41434-020-00192-z · Cited by 18

Multi-modification of food protein: Design, structural transformation, and functional application

Tang, W., Qu, J., Zhang, Y., Chen, J., Liu, W., He, J., & Liu, J. (2025) · Food Chemistry · doi:10.1016/j.foodchem.2025.146644 · Cited by 15

Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture

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559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus

Antoniani, C., Lattanzi, A., Pavani, G., Meneghini, V., Magrin, E., Weber, L., Porteus, M. H., Cavazzana, M., Mavilio, F., & Miccio, A. (2016) · Molecular Therapy · doi:10.1016/s1525-0016(16)33367-6

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

Traxler, E. A., Yao, Y., Wang, Y.-D., Woodard, K. J., Kurita, R., Nakamura, Y., Hughes, J. R., Hardison, R. C., Blobel, G. A., Li, C., et al. (2016) · Nature Medicine · doi:10.1038/nm.4170 · Cited by 366

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Métais, J., Doerfler, P. A., Mayuranathan, T., Bauer, D. E., Fowler, S., Hsieh, M. M., Katta, V., Keriwala, S., Lazzarotto, C. R., Luk, K., et al. (2019) · Blood · doi:10.1182/blood-2019-131318 · Cited by 2

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

Demirci, S., Leonard, A. K., Essawi, K., & Tisdale, J. F. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.010 · Cited by 56