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Case Study Of Thalassaem Talassaemia

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In collective view, genes are believed as the primary determinant of human health and they’re not modifiable. Thus, genetic disease are still considered as incurable disease. Until this day treatment and management strategies are mostly designed to improve only the particular signs and symptoms associated with those disorders. Although gene therapy become a promising treatment option for genetic diseases, the technique remains risky. Inserting gene into patients’ cells causes an imbalance in the whole living systems. Unwelcome immune response could cause serious illness or even death. Compared to other diseases, genetic diseases affect smaller population. Therefore, from the point of view of pharmaceutical companies, the high cost of developing a treatment makes it an unappealing prospect. A desire to bring a new hope for genetic diseases cure motivate me to pursue a PhD degree in System Biology, one of the programs in Harvard Integrated Life Sciences. …show more content…

The majority of patients, particularly in low-resource countries are children. In Indonesia, 300,000 children born with thalassemia each year, around 20 percent of them suffered from beta-thalassemia major, relying on blood transfusions all their lives. They spend an eight-hours process of transfusion every other week. Pre-meds those given on transfusion days caused them to become very drowsy and keep them from eating properly or functioning for basically an entire day.
There is imbalance between the number of patients and medical resources. Patients received no or minimal blood transfusions and iron chelator such as desferrioxamine is very expensive. The only cure for thalassaemia is stem cell transplantation. Unfortunately, not every countries have stem cell transplantation available and appropriate donor is hard to

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