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

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Article: e00253-20 | Published online: 03 Mar 2023

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Licínio Manco, Raquel Santos, Catarina Rocha, Luís Relvas, Celeste Bento, Tabita Maia, Verónica Gomes, António Amorim & Maria J. Prata. (2022) Hb F Levels in β-Thalassemia Carriers and Normal Individuals: Known and Unknown Quantitative Trait Loci in the β-Globin Gene Cluster. Hemoglobin 46:3, pages 168-175.
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Vigneshwaran Venkatesan, Abisha Crystal Christopher, Manuel Rhiel, Manoj Kumar K. Azhagiri, Prathibha Babu, Kaivalya Walavalkar, Bharath Saravanan, Geoffroy Andrieux, Sumathi Rangaraj, Saranya Srinivasan, Karthik V. Karuppusamy, Annlin Jacob, Abhirup Bagchi, Aswin Anand Pai, Yukio Nakamura, Ryo Kurita, Poonkuzhali Balasubramanian, Rekha Pai, Srujan Kumar Marepally, Kumarasamypet Murugesan Mohankumar, Shaji R. Velayudhan, Melanie Boerries, Dimple Notani, Toni Cathomen, Alok Srivastava & Saravanabhavan Thangavel. (2023) Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies. Molecular Therapy - Nucleic Acids 32, pages 671-688.
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Taqdees Arif, Aroosa Farooq, Fridoon Jawad Ahmad, Muhammad Akhtar & Mahmood S. Choudhery. (2022) Prime editing: A potential treatment option for β‐thalassemia. Cell Biology International 47:4, pages 699-713.
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Rosa Catapano, Raffaele Sessa, Silvia Trombetti, Elena Cesaro, Filippo Russo, Paola Izzo, Alexandros Makis & Michela Grosso. (2023) Identification and Functional Analysis of Known and New Mutations in the Transcription Factor KLF1 Linked with β-Thalassemia-like Phenotypes. Biology 12:4, pages 510.
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Udani Gamage, Kesari Warnakulasuriya, Sonali Hansika & Gayathri N. Silva. (2023) CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia. Thalassemia Reports 13:1, pages 51-69.
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Guoqiang Zhou, Haokun Zhang, Anning Lin, Zhen Wu, Ting Li, Xumin Zhang, Hongyan Chen & Daru Lu. (2022) Multi-Omics Analysis in β-Thalassemia Using an HBB Gene-Knockout Human Erythroid Progenitor Cell Model. International Journal of Molecular Sciences 23:5, pages 2807.
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