Genes coagulation disorders

Hemophilia A and B are coagulation disorders that result from defects in the genes encoding for plasma coagulation proteins. Hemophilia A (classic hemophilia) is caused by the deficiency of factor VIII, and hemophilia B (Christmas disease) is caused by the deficiency of factor IX. The incidences of hemophilia A and B are estimated at 1 in 5000 and 1 in 30,000 male births, respectively. Both types of hemophilia are evenly distributed across all ethnic and racial groups.1... 

Intracellular protein delivery is considered to be the most direct, fastest and safest approach for curing gene-deficiency diseases or disorders affecting primarily cellular processes, such as cystic fibrosis, coagulation disorders, otl-antitrypsin deficiency, immunoglobulin deficiencies, endocrine disorders and lysosomal storage diseases, where the expression of required proteins in the host cells is limited due to the defects in the required cellular (endogenous transcriptional) machinery/ ... 

Replacement of deficient gene products or even of organs is also utilized in the treatment of genetic disorders for example, replacement of coagulation factor VIII in hemophilia A, of or-antitrypsin in persons deficient in this factor or of pancreatic islet cells in some forms of diabetes mellitus. 

Approximately 80% of hemophilia patients have hemophilia A this is a congenital bleeding disorder resulting from insufficient levels of FVIII coagulation activity, and is characterized by a prolonged clotting time. Because the FVIII gene that codes for the FVIII protein is located on the X chromosome, virtually all clinically affected individuals are male [1]. 

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