Gene Therapy Weekly
Welcome to NewsRx!
Learn more about a six-week, no-risk free trial of Gene Therapy Weekly
We're a pay-per-view site for premium content. If you'd like to purchase this article, it's only $3.00.
Hemophilia B
Research on hemophilia b described by A.H. Chang and colleagues
December 18th, 2008
According to a study from the United States, "We have developed a lentiviral vector system for human factor IX (hFIX) gene transfer in hematopoietic stem cells (HSCs) that provides erythroid cell-derived systemic protein delivery following nonmyeloablative conditioning and in vivo methylguanine methyltransferase (MGMT) drug selection. After bone marrow transplantation under moderate Busulfan conditioning, the initial hFIX expression in the chimeras was minimally detectable." "However, the hFIX levels rose sharply following in vivo MGMT-drug selection and eventually reached a level that is considered curative in hemophilia B therapy (>500 ng/ml). The rise of hFIX...
Source: Gene Therapy Weekly (2008-12-18)
|
