What major problems must scientists overcome before gene therapy becomes a common technique for treating disease?
Scientists need to identify more efficient ways to deliver genes to the body. To treat cancer and other diseases effectively with gene therapy, researchers must develop vectors that can be injected into the patient and specifically focus on the target cells located throughout the body. More work is also needed to ensure that the vectors will successfully insert the desired genes into each of these target cells. Researchers also need to be able to deliver genes consistently to a precise location in the patient’s DNA, and ensure that transplanted genes are precisely controlled by the body’s normal physiologic signals. Although scientists are working hard on these problems, it is impossible to predict when they will have effective solutions. • The first disease approved for treatment with gene therapy was adenosine deaminase (ADA) deficiency. What is this disease and why was it selected? ADA deficiency is a rare genetic disease. The normal ADA gene produces an enzyme called adenosine deam
