Gene therapy Progress and Prospects: Gene therapy for the hemophilias

Gene Therapy for the Hemophilias

Commentary Gene therapy for the hemophilias

The hemophilias are a group of severe bleeding disorders resulting from the lack of functional blood coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Factor IX, when activated, catalyzes the conversion of factor X to its activated form, and activated factor VIII serves as a cofactor in this reaction. Clinically the disorders are indistinguishable, and they were not distinguis...

Gene therapy for PIDs: Progress, pitfalls and prospects

Substantial progress has been made in the past decade in treating several primary immunodeficiency disorders (PIDs) with gene therapy. Current approaches are based on ex-vivo transfer of therapeutic transgene via viral vectors to patient-derived autologous hematopoietic stem cells (HSCs) followed by transplantation back to the patient with or without conditioning. The overall outcome from all t...

Progress and Prospects of Anti-HBV Gene Therapy Development

Despite the availability of an effective vaccine against hepatitis B virus (HBV), chronic infection with the virus remains a major global health concern. Current drugs against HBV infection are limited by emergence of resistance and rarely achieve complete viral clearance. This has prompted vigorous research on developing better drugs against chronic HBV infection. Advances in understanding the...

Gene therapy: progress and predictions

The first clinical gene delivery, which involved insertion of a marker gene into lymphocytes from cancer patients, was published 25 years ago. In this review, we describe progress since then in gene therapy. Patients with some inherited single-gene defects can now be treated with their own bone marrow stem cells that have been engineered with a viral vector carrying the missing gene. Patients w...