Gene therapy for bleeding disorders

Curr Opin Hematol. 2002 Nov;9(6):511-5. doi: 10.1097/00062752-200211000-00008.

Abstract

The goal of gene therapy for bleeding disorders is to provide stable insertion and expression of a particular gene whose absence is responsible for a particular disease. The bleeding disorders for which the most basic and clinical research has been completed are the hemophilias factor VIII deficiency and factor IX deficiency. These X-linked diseases are caused by single-gene mutations; replacement of the defective gene has not only resulted in clinical and biochemical improvement in animal models but also provided promising results in phase I clinical trials. An ideal gene transfer approach to the treatment of hemophilia would require a minimally invasive procedure for gene delivery, have minimal associated morbidity, and result in long-term transgene expression, ideally yielding factor levels in the therapeutic range. Multiple approaches to gene transfer in the hemophilias are currently under investigation.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Blood Coagulation Factors / genetics
  • Clinical Trials as Topic
  • Coagulation Protein Disorders / therapy*
  • Genetic Therapy / methods*
  • Genetic Vectors / therapeutic use
  • Hemophilia A / therapy
  • Humans

Substances

  • Blood Coagulation Factors