use#LAA Gene Therapy of the b-Hemoglobinopathies by Lentiviral Transfer of the bA(T87Q)-Globin Gene
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Beta thalassemia gene therapy using lentiviral vectors
Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising ...
متن کاملGene Therapy in Thalassemia and Hemoglobinopathies
Sickle cell disease (SCD) and ß-thalassemia represent the most common hemoglobinopathies caused, respectively, by the alteration of structural features or deficient production of the ß-chain of the Hb molecule. Other hemoglobinopathies are characterized by different mutations in the α- or ß-globin genes and are associated with anemia and might require periodic or chronic blood transfusions. The...
متن کاملElucidation of βs/ Globin Gene clusters Haplotypes Related to Sickle Cell Anemia in Khuzestan Province, Southwest of Iran
Background & objectives: The researcher clarified that β/Globin gene cluster haplotypes in patients with sickle cell anemia provide useful population data as predictors of the disease severity, gene flow, and the origins of sickle cell mutation in this region. Materials and methods: A total of 150 subjects was investigated in two different groups for five polymorphism restriction site...
متن کاملEvaluation of the Efficacy of Lentiviral Vectors in Gene Therapy of Beta-thalassemia Patients: A Systematic Review
Background Beta thalassemiais a genetic blood abnormality identified through mutations, which reduce the synthesis of the ß-globin chain. Gene therapy through Lentiviral vectors have cured many of genetic disorders. The purpose of this study was to investigate the efficacy of lentiviral vectors in treatment of ß-thalassemia a...
متن کاملHIV-Derived Lentiviral Vectors: Current Progress toward Gene Therapy and DNA Vaccination
Lentiviral vectors are promising gene delivery tools capable of transducing a variety of dividing and non-dividing cells, including pluripotent stem cells which are refractory for transduction by murine retroviruses. Although there is a growing debate on the safety of lentiviral vectors for gene transfer, in particular for those derived from human immunodeficiency viruses, type one (HIV-1) and ...
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تاریخ انتشار 2016