Open AccessTreatment of Hemophilia With Genetic Engineering
Author(s) -
Axsom Kelly M.,
Manno Catherine S.
Publication year - 2004
Publication title -
transfusion alternatives in transfusion medicine
Language(s) - English
Resource type - Journals
eISSN - 1778-428X
pISSN - 1295-9022
DOI - 10.1111/j.1778-428x.2004.tb00117.x
Subject(s) - genetic enhancement , medicine , coagulation , gene transfer , factor ix , transgene , disease , gene , genetic disorder , bioinformatics , immunology , genetics , biology
Summary Hemophilia is a disorder characterized by decreased amounts of functional, circulating coagulation proteins VIII or IX. It has been well documented that by maintaining factor levels at ≥ 1% the major complications of severe disease, such as spontaneous bleeding, are averted. Successful gene transfer for hemophilia would result in persistent transgene expression at levels ≥ 1%. Hemophilia is an ideal candidate for the application of gene transfer because of the wide therapeutic window for the relevant factors, the availability of animal models, the ability of many tissues other than the liver to produce the coagulation proteins, and the desire for more convenient and cost‐efficient treatments. For these reasons, hemophilia has the potential to be one of the first severe genetic disorders to be treated, if not cured, with gene therapy. This paper summarizes the progress and challenges of gene transfer studies for hemophilia.