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Viability Of Gene Therapy For Hemophilia

2047 words - 9 pages

The Viability of Gene Therapy for Hemophilia

Gene therapy is a vast field, and experimentation is being attempted for a broad range of diseases. Gene therapy for hemophilia is specifically more viable than gene therapy for other diseases, even though it has some fallbacks of its own. Researchers have found that specific viral vectors can be used in gene therapy for hemophilia. Over the years, scientists have created a large experimental base by usage of both animal and human models. They have also found that there are certain technical problems to be overcome, despite the advances that have been made.

Gene therapy is the placement of a functional gene into the cells of one who is ...view middle of the document...

Additionally, the adenoviral vector has a large range of organisms that can host it. The adenoviral vector can also transduce, or influence the movement of genetic material, in target non-dividing cells especially the liver, which produces factor IX (High 396). The adenoviral vector system also has a high level of expression of the transgene, or a gene that is artificially placed into cells or the genome of an organism (Kaleko para 11). There are also several disadvantages to adenoviral vectors. There is only a short or medium length of expression of the transgene. Since adenoviral vectors generally target the cells of the liver, early generations of the vectors can cause damage to the liver. Also, antibodies can block the transduction of genetic material that the adenovirus controls (Kaleko para 11). The retroviral vector system also holds great potential for usage in gene therapy for hemophilia (Lemoine 216). The retroviral system has the possibility of long-term stability in terms of the transgene and the expression of the transgene. Also the retroviral system can integrate well into the DNA of a host cell. Additionally, there is a high chance of success for mutagenesis, or the mutation process in a cell, to occur upon insertion of the retrovirus into the organism (Kaleko para 11). If there is firm control of quality in terms of the viruses used, the risk of recombinant retroviruses causing harmful side effects, such as haphazard mutations, in both animals and humans is relatively low (Lemoine 216). On the other hand, there are also some downfalls to the usage of the retroviral vector system. The retroviruses can only transduce in cells that are not dividing. Also the virus vectors can be subject to deactivation by the DNA promoter sequence in cells (Kaleko para 11).
Gene therapy for hemophilia has specific characteristics that allow it to be more viable than gene therapy for other diseases. The transgenes do not need to be placed in a specific tissue because clotting factors can be produced in several cell types. While the liver generally produces clotting factors, myoblasts, fibroblasts and endothelial cells can also produce clotting factors. Other hereditary diseases such as sickle cell disease and thalassemias required tissue specific expression for a beneficial effect to occur in patients (High 389). Additionally, there does not have to be an exact amount of transgene placed into the cells of the patient. Patients who have a severe form of hemophilia have one percent or lower of normal clotting factor levels. Those who have a milder form of hemophilia have five percent of normal clotting factor levels only sporadically have spontaneous episodes of bleeding. Therefore, different amounts of transgene placed in a patient’s cells can still allow the patient to undergo a considerable phenotypical improvement. Finally, there are several animals which model the disease in the same manner in which the disease appears in humans. This allows...

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