Review Articles
Vol. 16 No. 1 (2024): Review Articles, Original Article, Scientific Letter, Case Reports Letter to the Editor
THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES
Gene therapy for Hemoglobinopathies.
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All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Published: August 31, 2024
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In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent b-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding b-globin in defective CD34 cells; more recently, gene editing techniques have been used involving either CRISPR-Cas9, transcription activation-like effector protein nuclease, zinc finger nuclease, and base editing to either induce fetal hemoglobin production at therapeutic levels or to genetically repair the underlying molecular defect causing the disease.
Here, we review recent gene editing studies that have started the development of a new era in the treatment of hemoglobinopathies and, in general, monoallelic hereditary diseases.
Keywords: Gene editing; gene therapy; Hemoglobinopathies; Thalassemia; Sickle Cell Anemia.
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How to Cite
“THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES: Gene therapy for Hemoglobinopathies”. (2024) Mediterranean Journal of Hematology and Infectious Diseases, 16(1), p. e2024068. doi:10.4084/MJHID.2024.068.
Copyright (c) 2024 Ugo Testa, Giuseppe Leone, Prof. M.D. Cappellini
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
