Lenti in red: progress in gene therapy for human hemoglobinopathies

Lenti 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 ...

Gene 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...

Progress toward human gene therapy.

Designing an Engineered Construct Gene Sensitive to Carbohydrate In-vitro and Candidate for Human Insulin Gene Therapy In-vivo

Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...

Designing an Engineered Construct Gene Sensitive to Carbohydrate In-vitro and Candidate for Human Insulin Gene Therapy In-vivo

Background and Aim: Diabetes is a common disorder worldwide, and exhaustive efforts have been made to cure this disease. Gene therapy has considered as a potential curative method that has more stability in comparison with the other pharmaceutical methods. However, the application of gene therapy as a definitive treatment demands further investigation. This study aim is to prepare a suitable hi...