CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia

CRISPR Gene Therapy: A Promising One-Time Therapeutic Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia

β-Thalassemia is an inherited hematological disorder that results from genetic changes in the β-globin gene, leading to the reduced or absent synthesis of β-globin. For several decades, the only curative treatment option for β-thalassemia has been allogeneic hematopoietic cell transplantation (allo-...

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Bibliographic Details
Main Authors: Udani Gamage, Kesari Warnakulasuriya, Sonali Hansika, Gayathri N. Silva
Format: Article
Language:English
Published: MDPI AG 2023-02-01
Series:Thalassemia Reports
Subjects:
β-thalassemia
β-globin
γ-globin
BCL11A
CRISPR/Cas9 technology
CTX001 therapy
Online Access:https://www.mdpi.com/2039-4365/13/1/6

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