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Iranian Journal of Pediatric Hematology and Oncology، جلد ۱۲، شماره ۱، صفحات ۵۵-۶۷
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عنوان انگلیسی Therapeutic approaches in patients with β-thalassemia
چکیده انگلیسی مقاله Beta-thalassemia (β-thal) is a congenital hemoglobinopathy explained by a decreased level (β+) or absence (βο) of β-globin gene expression. Microcytic hypochromic anemia and various clinical symptoms comprising severe anemia to clinically nonsymptomatic features. Treatment with an ordered blood transfusion and iron chelator agents can decrease transfusion iron overload that causes normal maturation. These patients also are at high risk for secondary iron overload because of erythropheron (GF15–TWSG1) release from erythroblasts resulting in erythroid hyperplasia. Based on the previous studies, chemicals such as hydroxyurea and 5-azacytidine are useful in treating β-hemoglobinopathy, including β-thal and sickle cell disease (SCD). Regarding both side effects and lifelong treatment of these chemical components, researchers have recently regarded gene-based treatments. These techniques, such as micro RNA gene silencing, viral-mediated gene editing, and clustered regulatory interspaced short palindromic repeats (CRISPR)-CAS9 systems, are the most commonly used gene therapy methods. Nowadays, ɣ-globin (fetal globin) gene reactivation is one of the most popular treatments for β-thal. Researches showed that these gene modification methods for γ-globin gene reactivation are also useful in increasing hemoglobin F (HbF) and helping patients with β-thal. In this review study, new therapeutic approaches to manage this disorder are regarded.
کلیدواژه‌های انگلیسی مقاله Beta-thalassemia, Genetic therapy, Hemoglobinopathy
نویسندگان مقاله | Mohammad Eini
Department of Hematology, faculty of paramedical, Birjand University of Medical Science, Birjand, Iran


| Mohammad Shoae
Department of Medical Biotechnology, Faculty of Medicine, Birjand University of Medical Sciences, Birjand, Iran


| Ebrahim Miri-moghaddam
Cardiovascular Diseases Research Center, Department of molecular medicine, Birjand University of Medical Sciences, Birjand, Iran
نشانی اینترنتی http://ijpho.ssu.ac.ir/browse.php?a_code=A-10-817-1&slc_lang=en&sid=1
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