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Gene Therapy Hemophilia

stance that stimulates blood vessel growth to damaged heart tissue and in this trial there was much success noted.The beginning of Gene Therapy began in the late 1980s, which was completely unsuccessful. In the fall of 1999 the death of University of Pennsylvania trial participant, Jesse Gelsinger was followed by much public outcry and legal problems that put an immediate halt on all gene therapy research. The reason Jesse was being treated with Gene therapy was to attempt to cure the teenagers rare liver disease. In the long run, the outcome of a government investigation concluded that researchers had not followed the rules. The FDA had all research put to a stand still until an appropriate monitoring and reporting system had been approved and was in place.As you can guess, each of these gene therapy procedures was first tested on animals. First, the virus, hemophilia B was injected into the dogs in the form of a virus. Then the dogs were treated with gene therapy and given the appropriate missing genes and adding them to muscle cells, which would accept the genes, and then produce the proper clotting factor and the clotting of the dogs blood would be about back on track. The result show that the blood-clotting times decreased from more than on hour down to about 15-20 minutes where as a normal dogs blood-clotting time is approximately six minutes. No side effects or problems were reported as a result of the treatment. Researchers at Stanford University School of Medicine had a similar outcome when they experimented, first on mice, then on dogs as well. (The process here shows how the new gene or foreign DNA is added into the nucleus of the chromosome with the help of the virus, represented here as AAV Vector)Gene therapy that treats hemophilia has been an increasingly popular method to take cure hemophilia A and B. Hemophilia A is the more common form that is caused by a missing gene used as a blood-clotting factor. A single gene int...

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