Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2008 Mar;140(5):479-87.
doi: 10.1111/j.1365-2141.2007.06942.x.

Gene therapy for haemophilia

Samuel L Murphy  1 , Katherine A High
Affiliations
Review

Gene therapy for haemophilia

Samuel L Murphy et al. Br J Haematol. 2008 Mar.

Abstract

The ultimate goal of gene therapy is the replacement of a defective gene sequence with a corrected version to eliminate disease for the lifetime of the patient. This challenging task is not yet accomplished, however significant progress is evident. An initial spate of clinical trials attempting the treatment of haemophilia with gene transfer primarily resulted in the demonstration of good safety profiles, but without efficacy. Subsequent reengineering of vector plasmids and delivery systems resulted in markedly improved outcomes in animal models of the disease. The most recent clinical trial for the treatment of haemophilia B with gene transfer showed transient achievement of efficacy in the highest dose cohort tested, but also exposed a previously hidden barrier to the future success of these treatments. The progress and problems of gene therapies for haemorrhagic disorders will be discussed. This review will concentrate on approaches in or near clinical application.

PubMed Disclaimer

References

    1. Andrews JL, Shirley PS, Iverson WO, Sherer AD, Markovits JE, King L, Lyons RM, Kaleko M, Connelly S. Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector. Human Gene Therapy. 2002;13:1331–1336. - PubMed
    1. Arruda VR, Hagstrom JN, Deitch J, Heiman-Patterson T, Camire RM, Chu K, Fields PA, Herzog RW, Couto LB, Larson PJ, High KA. Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood. 2001;97:130–138. - PubMed
    1. Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood. 2005;105:3458–3464. - PMC - PubMed
    1. Blacklow NR, Hoggan MD, Kapikian AZ, Austin JB, Rowe WP. Epidemiology of adenovirus-associated virus infection in a nursery population. American Journal of Epidemiology. 1968;88:368–378. - PubMed
    1. Blacklow NR, Hoggan MD, Sereno MS, Brandt CD, Kim HW, Parrott RH, Chanock RM. A seroepidemiologic study of adenovirus-associated virus infection in infants and children. American Journal of Epidemiology. 1971;94:359–366. - PubMed

Publication types