Just recently a group of scientists tested a type of gene therapy that can put an end to sickle cell anemia and beta thalassemia. The newly developed gene has a healthy beta globin gene that promotes a cell's hemoglobin production. With this new gene, the cells that were lacking this beta globin gene or have a non functioning can now hope to produce hemoglobin. And this type of therapy would bear few risks due to the fact that this new gene can be tested on a blood sample before it's actually given to you. By giving these new cells a functioning beta globin gene this can lead to the cure of beta thalassemia. The success rate in a process of gene therapy can be quite difficult though because when the gene is injected it will randomly place itself, meaning the gene could not be expressed, so an insulator is made to lure the gene into the genome area increasing the success rate of gene therapy.
With these new findings maybe it can be possible to master gene therapy and be able to replace any dysfunctional gene with a functional one. This can lead to the cure of many genetic disorders such as PKU.
Posted by Khoa Chu (A)