What are the advantages and disadvantages of gene therapy for haemophilia?

The use of gene therapy for the treatment of haemophilia is in many ways a promising option, but also has some disadvantages. On the pro -side, haemophilia is an ideal genetic disorder to treat in this way because it occurs due to a single genetic defect. This type of treatment can be considered successful, although it has only a minimal increase in blood coagulation factors, as this is still a huge benefit for patients. On the other hand, the results were inconsistent in clinical trials with human test entities. There is also a risk that the patient's body will develop antibodies to viral vectors used to supply genetic material to cells. When it comes to genetic disorder, it is quite simple, caused by one known genetic defect that limits the body's ability to generate blood factors. Unlike other genetic diseases that may include numerous defects, relaylielielies are easy to target gene therapy.

Another reason why gene therapy of haemophilia is so promising that even minimal success from the procedure can mean a significant improvement in the quality of life in patients. In some tests, therapy led to a slight increase in blood coagulation factors, significantly below normal, but the time that took the clot of blood to decrease significantly. People who see such improvements can be able to go for a long time without additional infusions of the clotting factor.

However,

gene therapy for haemophilia is not positive because the results of treatment are rather inconsistent. Many animal studies have been conducted, often with good results, but this was not translated into similar results in human beings. Different methods of delivery through different types of viral vectors, as well as different doses, led to mixing results, with a consistently successful method of treatment that still shows ineffectable.

Treatment method is another con in terms of gene therapy forHemophilia. In order to obtain genetic material into the cells, it must be attached to viruses, which are then injected into the body so that these cells can attack. In some cases, this may cause an immune response in the patient. His body can then produce antibodies that destroy viruses and prevent treatment from occurring.