Treatment of bubble boy syndrome with Gene therapy

Report has been published previously with regards to disorder of children on the functioning of their immune system. A well known gene therapy in advanced form is discovered to treat children with fatal genetic disorder. Many children with X link have combined X-SCID as well as immune deficiency stays in bubble for getting a protection.

Trial has been conducted where healthy version of faulty gene has been provided to nine of the baby boys. Just after the treatment has been conducted, eight such boys were seen to stay alive after 43 months of treatment.

The experiment has been so successful that those baby boys are really capable to live a healthy life without being isolated who otherwise were kept in a virtual bubble or sterile room. They were kept under constant medication to prevent infections.

Bubble boy syndrome is an immune disorder that is affected to small boys. The disease is really rare and is affected to 1 out of 100,000 people. The parents need to keep the baby really safe so that he stays away from any other types of infections. The patient need to stay inside a tube or a plastic pod where he needs to consume sterilized food and wear sterilized clothes. Even everyday germs can be fatal to these types of patients. They do suffer from the X linked immune deficiency. The medical science has taken their hands up in treating these types of patients. But today a therapy named as gene therapy is likely to get these patients a perfect cure.

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According to the recent report from Prof Adrian Thrasher Great Ormond street hospital, The disease named bubble boy syndrome affects only one among 2,50,000 children in United Kingdom. Also the clinical trial was led by the scientists in France, US as well as Britain where genes were transferred into the children with X-SCID.

Safe therapy

Virus was used by the therapy in order to transfer the gene but in exchange people with other genes leads to leukemia which has a number of 5 out of 20 patients. Thus, the scientist took the initiative of changing the virus of the experiment which does not switch on the neighboring genes in a patient.

Bone marrow was extracted in the boys who are having an age of less than 12 months. There after the virus was found out that carries the healthy genes and then transferred accordingly. The study also says that, a boy died due to the serious infection when the gene therapy was conducted.

A meridian average was found out where the particular therapy applied which resulted into living persistence of eight out of nine boys.  Even after the therapy was applied, leukemia was not applied. But, the risk of cancer is not eliminated completely.

Best leaders associated with the project

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As compared to the existing option, the gene therapy so discovered was much more effective which is also known to be a gentle procedure. This procedure also involves transplantation of the bone marrow from the donors who are compatible. But, it is really very hard to find out. After implementation of this process, the survival rate was around 85 to 90 percent.  But, the percentage can also fall down to 50 if the children have infection at the time of transplant.  Chemotherapy is also required during the process of transplantation as this helps patient to accept the changes. But, some patients can also have an unpleasant side effect.

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Other facts about the disease

According to Prof Thrasher, It is quite natural to admit the patient in hospital if he is sick. Even if the infection stays for the period of two to three months, he stays in hospital. Inversely, if the patient is fit, he can be out from hospitals in just few days. Professor also added that, there will be good implications with regards to the treatment for the diseases like thalassaemia, inherited blood disorder, metabolic syndrome and sickle cell anemia.

According to a professor in the University of Oxford, the best leaders in the field worked in the project.  It was also the pan world effort.  It will be altogether a big step if the treatment does not include chemotherapy. This therapy has got a great impact on people today who have children with such disorder.

Discovery of gene therapy

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The gene therapy that was taken up earlier was capable of keeping the patient alive. But, people are afraid to adopt it as this has a side effect to the patient. Yes, leukemia is affected to the patient suffering from bubble boy syndrome. It is just due to the fact that the earlier variation of gene therapy had the capacity to restore only some specific variation of immune cells. Thus, the patient seemed not to have fruitful cure. Thus, they would require injections that fight with pathogen.

New viral vehicle

The researchers went through more experiments in order to overcome. They adopted new viral vehicle in this situation. It is a lab grown variation of HIV that is associated with risky innards torn out. Here the bone marrow of each patient is extracted and inserts the healthy stem cells. There is a vast difference between the old stem cell therapy and new therapy as the new one does not require total cycle of reproduction. Thus, the healthy gene is inserted in more stem cells. Thus, reinjection can be done to the patient suffering from the bubble boy syndrome. Doctors also have prepared the bone marrow before time for the 5 patients. More studies would be conducted where it should be found out whether the new gene therapy will be used in much easy and effective way.

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