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Genome Editing for β-Hemoglobinopathies: Advances and Challenges

Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, Université de Paris, INSERM UMR 1163, F-75015 Paris, France
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Academic Editor: Marina Cavazzana
J. Clin. Med. 2021, 10(3), 482; https://doi.org/10.3390/jcm10030482
Received: 16 December 2020 / Revised: 19 January 2021 / Accepted: 25 January 2021 / Published: 28 January 2021
(This article belongs to the Special Issue New Frontiers in Molecular Hematology)
β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a compatible donor for allogeneic bone marrow transplantation, the lifelong therapeutic options are symptomatic care, red blood cell transfusions and pharmacological treatments. The last decades of research established lentiviral-mediated gene therapy as an efficacious therapeutic strategy. However, this approach is highly expensive and associated with a variable outcome depending on the effectiveness of the viral vector and the quality of the cell product. In the last years, genome editing emerged as a valuable tool for the development of curative strategies for β-hemoglobinopathies. Moreover, due to the wide range of its applications, genome editing has been extensively used to study regulatory mechanisms underlying globin gene regulation allowing the identification of novel genetic and pharmacological targets. In this work, we review the current advances and challenges of genome editing approaches to β-hemoglobinopathies. Special focus has been directed towards strategies aimed at correcting the defective β-globin gene or at inducing fetal hemoglobin (HbF), which are in an advanced state of clinical development. View Full-Text
Keywords: genome editing; β-hemoglobinopathies; gene therapy genome editing; β-hemoglobinopathies; gene therapy
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MDPI and ACS Style

Frati, G.; Miccio, A. Genome Editing for β-Hemoglobinopathies: Advances and Challenges. J. Clin. Med. 2021, 10, 482. https://doi.org/10.3390/jcm10030482

AMA Style

Frati G, Miccio A. Genome Editing for β-Hemoglobinopathies: Advances and Challenges. Journal of Clinical Medicine. 2021; 10(3):482. https://doi.org/10.3390/jcm10030482

Chicago/Turabian Style

Frati, Giacomo, and Annarita Miccio. 2021. "Genome Editing for β-Hemoglobinopathies: Advances and Challenges" Journal of Clinical Medicine 10, no. 3: 482. https://doi.org/10.3390/jcm10030482

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