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Gene therapy: The safety and efficacy of this treatment on hemophilia B

Research Question:
Should gene therapy be used to treat hemophilia B compared to other treatments?
Hypothesis:
Gene therapy increases the chances of curing hemophilia B more than other treatments.
Background/History of the Issue:
Gene therapy, first introduced in the 1960s, has been a controversial form of treatment for some
time. Many studies have experimented with it, but so far it hasnt been definitively proven a safe
and effective treatment. Gene therapy is an experimental technique of utilizing DNA to combat
defective and diseased genes. It can be done via an injection or an IV. To inject the gene
treatment into the patient, the gene is first often carried in viruses, so that they can be delivered
to the cell through infection. Gene therapy also carries implications for genetic modification, as
genes can be inserted to zygotes as well as somatic cells, which would lead to designed traits in
offspring. This heavy societal implication has delayed more exploration on the subject, but
progress is still continuing and many studies have been made experimenting on the possible
successful cures this treatment technique can provide. These studies have mostly been conducted
on genetic diseases, but experiments on how scientists are also studying how this technique
could affect cancer cells.
Rationale:
I believe this is an important topic because if gene therapy works correctly, it will signify
enormous scientific and technological advancements for contemporary society. The implications
of this would change methods of scientific research and impact the world as it progresses. By
focusing on hemophilia B, I can explore the effect it specifically has on genetic diseases.

Furthermore, by comparing my research on gene therapy to currently used treatments, I can


analyze the benefits and detriments of hemophiliac patients options, and draw a concrete
conclusion on their efficacy with a recommended solution.
Basis of Hypothesis:
My hypothesis focuses on the effect gene therapy has on hemophilia B because studies show this
specific area to show the most promising results. Gene therapy may eventually provide the
solution for all genetic diseases. This is because genetic diseases can be corrected in this process
simply by inserting normal genes to replace the defectives. In the case of hemophilia B, there is a
lack of factor IX proteins which clot blood, but through gene therapy they are supplied
permanently. Therefore, by focusing on hemophilia B, a genetic disease, I can create a basis to
hypothesize for all genetic diseases. I am interested in viewing the different aspects of the
treatment, to find the overall best solution. To collect data, I can conduct meta analysis to gather
sources and data and create one comprehensive conclusion through other research experiments.
Additionally, throughout my research themes have emerged in which predict that gene therapy is
worth the risk, supporting my hypothesis that gene therapy should be implemented as sanctioned
hemophilia B treatment.
Operational Definition:
Gene therapy: An experimental technique that treats or prevents disease through inserting genes,
it is a recent advancement in science which has controversial results.
Hemophilia B: A genetic disease, it is caused by lack of factor IX production, resulting in lack of
blood clots.
Descriptors Used for Literature Search:
Gene therapy and hemophilia B
Gene therapy process

Hemophilia B treatments
Effects of gene therapy