Gene Therapy for β-Hemoglobinopathies
نویسندگان
چکیده
منابع مشابه
Customizing the genome as therapy for the β-hemoglobinopathies.
Despite nearly complete understanding of the genetics of the β-hemoglobinopathies for several decades, definitive treatment options have lagged behind. Recent developments in technologies for facile manipulation of the genome (zinc finger nucleases, transcription activator-like effector nucleases, or clustered regularly interspaced short palindromic repeats-based nucleases) raise prospects for ...
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 کاملLenti in red: progress in gene therapy for human hemoglobinopathies.
Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic beta-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of ...
متن کاملGene Therapy for β - Thalassemia
Gene transfer for β-thalassemia requires gene transfer into hematopoietic stem cells using integrating vectors that direct regulated expression of β globin at therapeutic levels. Among integrating vectors, oncoretroviral vectors carrying the human βglobin gene and portions of the locus control region (LCR) have suffered from problems of vector instability, low titers and variable expression. In...
متن کاملGene therapy approaches to the treatment of hemoglobinopathies Review
Hemoglobinopathies such as thalassemia and sickle cell anemia are potentially amenable to gene therapy. Applicable gene therapy strategies can be divided into four categories: those that replace the faulty gene with a complete transcript ional unit , those that activate transcription of fetal hemoglobin genes, those that modify the endogenous gene itself , and those that attempt to repair the d...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2017
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2017.03.024