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 suffering from a disease in order to give a new
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They can be produced in higher titer, the concentration of an antibody that is determined by the amount of dilution before it no longer reacts to antigen affirmatively. 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