Marjan Raie,1Saman Hakimian,2,*
1. Baclocher student of Microbiology Islamic Azad University , Rasht Gilan , Iran 2. M.sc student of Microbiology Islamic Azad University Central Tehran Branch,Iran,Tehran
Introduction: Introduction:
Nowadays around the world gene therapy is being developed and the most common diseases such as cancer can be treated with gene therapy.
According to the U.S. Food and Drug Administration (FDA)، gene therapy is often referred to as a product that inserts a healthy gene into the patient's body by the appropriate vector to affect the target cells as designed.
In the last three decades, hemophilia A and B have been promoted as disorders of gene therapy.
Hemophilia A and B are inherited diseases that are dependent on the sex chromosome X that cause clotting disorders. Hemophilia A is caused by mutations in F8C genes that cause deficiency or impairment of factor 8 function. And hemophilia B is caused by mutations in F9 genes that cause deficiency and disorders or factor 9.
Methods: Material methods:
Hemorrhage in hemophilia can be undamaged; these bleeds often happen in joints and in muscle tissues. If hemophilia is very severe, with normal activities, bleeding may occur spontaneously, But people with milder hemophilia may occur with trauma.
Here's how to treat hemophilia with the help of gene therapy. One of these methods is done with Adeno associated virus.
Adeno Associated virus (AAV) is a small virus that has a DNA and because of its good specification، it is used as vector in gene engineering and gene therapy. One of the advantages of this vector is that it is not pathogenic and protein has a coating that binds the cell and enters the gene into the cell.
Gene therapy with AAV simply means that we apply healthy genetic material، the F9 and F8 genes، into the vector
of the adeno associated virus that we remove viral genes but not end of both sides of DNA , because contains viral information which is necessary for the high level of expression of the healing gene that vector carries, now This vector is injected into the body and targets the liver cells.
Another method of treatment hemophilia B is Etranacogene Dezaparvovec.
Results: Results:
Tissue factor pathway inhibitor (TFPI ) is an endogenous inhibitor of the outer coagulation pathway.
Generally, in hemophilia A and B patients, TFPI inhibition is an alternative therapeutic approach that increases the external coagulation pathway.
Conclusion: Conclusion:
An experiment has been conducted that assesses the prevalence of anxiety disorders in hemophilia patients who have injected the factor and their families.
100 groups of two hemophilic patients in the age group of 8-15 years who underwent regular coagulation factor injections And their parents were compared to 100 groups of parents whose children were undergo control but not suffering.
The prevalence of anxiety was 32% in hemophilia and 16% among the control group.
Distress among parents of adolescents with hemophilia was 45% and 24% in control group.
Observations indicate that anxiety was higher in older patients and in patients with lower gaps in transmitting coagulation factors
As a result، hemophilia patients and their parents are at higher risk of developing anxiety disorders than others.