Positive outcomes have been shown in many patients who undergo this procedure. However, bone marrow transplantations are very high risk. The vector used in this procedure is an adeno-associated virus, as described previously. Using a virus as the vector in gene therapy is termed transduction. In using gene therapy technique on thalassemia patients, expression is an important issue.
Expression is the term for the characteristics that the gene exhibits.
Not only expression is an issue but sustained expression is an essential point for success of the procedure. A few key factors must be met for proper expression for thalassemia patients. Second, the degree of expression is important. If all of these elements come together, the use of gene therapy for those with thalassemia will have favorable results. At this point in the management of thalassemia, gene transfer therapy is the route that most professionals look to for the future.