Gene therapy for hemophilia A

Abstract

Abstract: Hemophilia A is an X chromosome-linked recessive inheritable bleeding disorder with an estimated incidence of 1-2 per 10000 males. Patients with Hemophilia A experience spontaneous bleeding into joints, soft tissues and other sites. Uncontrolled haemorrhagic episodes are crippling and potentially life threatening. Hemophilia A is caused by a deficiency or abnormality in factor Ⅷ, which is one of the most attractive candidates for gene therapy. In this article, the research of vectors and target cell in gene therapy for hemophilia A was reviewed.

Key words: hFⅧ, Hemophilia A, Gene therapy

CLC Number:

R554⁺.1

LIU Mo-jun, LIU Yan-ping, LIU Xiong-hao. Gene therapy for hemophilia A[J]. Chinese Journal of Clinical Pharmacology and Therapeutics, 2010, 15(8): 932-937.

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