Article
Phenotypic correction and stable expression of factor VIII in hemophilia A mice by embryonic stem cell therapy.
Genetics and molecular research : GMR - 13 May 2013
Wang J J, Kuang Y, Zhang L L, Shen C L, Wang L, Lu S Y, Lu X B, Fei J, Gu M M, Wang Z G
Abstract excerpt
Hereditary deficiency of factor VIII (FVIII) leads to hemophilia A, a severe X-linked bleeding disorder. Current therapies include fixed-dose FVIII prophylaxis, factor replacement therapy, and most recently, gene therapy. Prophylaxis and FVIII replacement therapies are limited by incomplete efficacy, high cost, restricted availability, and development of neutralizing antibodies in chronically treated individuals....
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