Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia.

Recent progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. Curr Drug Targets. 2019 Jul 26;: Authors: Hamed EM, Meabed MH, Aly UF, Hussein RRS Abstract Thalassemia syndromes are a group of inherited blood disorders caused by limitation or absence of alpha or beta- globin chain synthesis resulting in ineffective erythropoiesis and chronic hemolytic anemia. According to the clinical fact of thalassemia as recessive autosomal, thalassemia divided into alpha and beta thalassemia. The main complication of beta thalassemia is excessive red blood cells destruction, oxidative stress, extramedullary hematopoiesis pseudotumors, hemosiderosis-induced organ dysfunction, malignancy, polyneuropathy, myopathy, osteoporosis, and leg ulcers. Although, the first obligatory therapy for thalassemia major is a blood transfusion, cause cardiac and hepatic hemosiderosis that is the most common cause of death in these patients. The targeted sequencing protocols include Hb F inducers, ineffective erythropoiesis correctors, antioxidant, vitamins, and herbals. Resveratrol is a novel herbal therapeutic approach for β-thalassemia. New appearance of molecular therapy in response to adverse effects of drugs. ActRIIB/IgG1 Fc recombinant protein is a novel molecular therapy in thalassemia. This review focuses light on maximizing the efficacy of unique combinations and new future therapeutic interventions in thalassemia that rever...
Source: Current Drug Targets - Category: Drugs & Pharmacology Authors: Tags: Curr Drug Targets Source Type: research