THERAPEUTIC GENE EDITING FOR HEMOGLOBINOPATHIES

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 v...

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Bibliographic Details
Main Authors: Ugo Testa, Giuseppe Leone, Maria Domenica Cappellini
Format: Article
Language:English
Published: PAGEPress Publications 2024-08-01
Series:Mediterranean Journal of Hematology and Infectious Diseases
Subjects:
Online Access:https://www.mjhid.org/mjhid/article/view/5768
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