PMID: 9558383May 23, 1998Paper

Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy.

Blood
S ConnellyM Kaleko

Abstract

Hemophilia A is caused by a deficiency of blood coagulation factor VIII (FVIII) and has been widely discussed as a candidate for gene therapy. While the natural canine model of hemophilia A has been valuable for the development of FVIII pharmaceutical products, the use of hemophiliac dogs for gene therapy studies has several limitations such as expense and the long canine generation time. The recent creation of two strains of FVIII-deficient mice provides the first small animal model of hemophilia A. Treatment of hemophiliac mice of both genotypes with potent, human FVIII-encoding adenoviral vectors resulted in expression of biologically active human FVIII at levels, which declined, but remained above the human therapeutic range for over 9 months. The duration of expression and FVIII plasma levels achieved were similar in both hemophiliac mouse strains. Treated mice readily survived tail clipping with minimal blood loss, thus showing phenotypic correction of murine hemophilia A by in vivo gene therapy.

Citations

Jan 14, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ekaterina BreousJames M Wilson
Jan 5, 1999·Haemophilia : the Official Journal of the World Federation of Hemophilia·S Connelly, M Kaleko
Dec 7, 2006·Proceedings of the National Academy of Sciences of the United States of America·Anna AronovichYair Reisner
Sep 1, 1999·Proceedings of the National Academy of Sciences of the United States of America·T VandenDriesscheM K Chuah
Oct 27, 1999·Proceedings of the National Academy of Sciences of the United States of America·M BurtonL Couto
Mar 31, 1999·Proceedings of the National Academy of Sciences of the United States of America·L WangI M Verma
May 3, 2003·The Journal of Clinical Investigation·Federico MingozziRoland W Herzog
Jul 11, 2006·The Journal of Clinical Investigation·Qizhen ShiRobert R Montgomery

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Blood Clotting Disorders

Thrombophilia includes conditions with increased tendency for excessive blood clotting. Blood clotting occurs when the body has insufficient amounts of specialized proteins that make blood clot and stop bleeding. Here is the latest research on blood clotting disorders.