Review Article
Year: 2020 | Month: July-September | Volume: 5 | Issue: 3 | Pages: 158-173
Gene Therapy for B-thalassemia; New Challenges
Maged MN1, Mohamed MN2, Lamia H. Shehata3
Mazahmiya Hospital1, Ministry of Health, Kingdom of Saudi Arabia, Department of ob /gyn ,
King Fahd Hospital2, Ministry of Health , Kingdom of Saudi Arabia, Department of Surgery ,
Care National hospital3, Department of Radiology.
Corresponding Author: Maged MN
ABSTRACT
Recently, gene therapy clinical preliminaries have been effectively applied to hemoglobinopathies, for example, sickle cell disease (SCD) and β-thalassemia. Among the extraordinary disclosures that prompted the structure of genetic ways to deal with fix these disorders is the revelation of the β-globin locus control region and a few related transcription factors, which determine hemoglobin exchanging just as significant level, erythroid-specific expression of genes at the ß-globin locus. Additionally, expanding proof shows that lentiviral vectors are proficient tools to embed large DNA components into nondividing hematopoietic stem cells, indicating consoling safe coordination profiles. On the other hand, genome altering could reestablish expression of fetal hemoglobin or target explicit mutations to restore expression of the wild-type β-globin gene. The latest clinical preliminaries for β-thalassemia and SCD are demonstrating promising results: patients had the option to stop transfusions or had diminished transfusion necessities. Be that as it may, toxic myeloablation and the significant expense of current ex vivo hematopoietic stem cell gene therapy stages speak to a barrier to a far reaching use of these methodologies. In this review, we sum up these gene therapy procedures and progressing clinical preliminaries. At last, we talk about potential systems to improve results, lessen myeloablative regimens and future difficulties to decrease the cost of gene therapy platform.
Keywords: gene therapy, β-thalassemia, hemoglobinopathy