Imagine living a life where you are unable to do simple things like practice a sport or even walk without being afraid of hurting yourself. This is the life of people suffering from a blood condition known as hemophilia. People who suffer from this risk dying even from a small cut.
The Benefits Gene Therapy Might Bring to Hemophilia Patients
Hemophilia is a disease caused by the lack of a gene which makes the protein needed for blood to clot. This means that from a cut, people with hemophilia can bleed out if they don’t get treatment.
Gene therapy has already helped ten men by stopping all symptoms related to hemophilia. These were part of an experimental gene therapy treatment that set out to treat hemophilia. The study results are giving hope to people who suffer from the condition as it might help them lead a normal life by getting rid of the disease.
Hemophilia was noted to affect mostly men.Those that suffer from it need to prevent severe bleeding more than once a week. The gene therapy was able to supply the missing protein. Researchers used a modified virus which doesn’t cause other diseases. The virus triggers the DNA in liver cells to make them start and carry out the clotting process.
This gene therapy treatment was provided through an IV. In the study, which was published in the New England Journal of Medicine, all of the ten men who received the therapy now have an average range clotting factor. Bleeding episodes were reduced from one a month to less than one a year. 9 out of these 10 men don’t need any other clotting treatments.
None of these men presented any serious side effects. The follow-up is still too short to tell if the gene therapy is going to last long enough though. This gene therapy is still experimental and its total costs have yet to be calculated. The clotting factor treatment could cost up to $200,000 per year for every patient, but could also help a lot of people.
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