Despite trying to maintain a professional level of emotional

Despite trying to maintain a professional level of emotional distance, you have become attached to Lee and his good natured family. You spend evenings searching the medical literature for potential treatments including clinical trials, to help them. You remember learning about gene therapy trials for SCID (Severe Combined ImmunoDeficiency) and LCA (Leber\'s Congenital A maurosis a type of hereditary blindness in the New England Journal of Medicine. How would you describe the main goal of gene therapy to this family? Several different types of viruses, including adenoviruses, have been used as vectors for gene therapy in humans. Why are viruses used for gene therapy? The two basic types of gene therapy are ____ and _____. What is the main difference between them? Which type would best help this patient and his family? Why? What types of risks/problems are currently associated with gene therapy?

Solution

1. The main goal of gene therapy is to correct genetic diseases by inserting genes from a normal person to cover for the damaged or different type of genes present in the patient\'s body.

2. Viruses are used for gene therapy because they are adept at integrating their DNA with host DNA, and they can increase in number in the host if wanted. They have smaller DNA than most organisms, which is well studied and can be modified to insert foreign DNA.

3. Somatic - to correct the genetic diseases in the body to cure it\'s symptoms, while not making the subsequent generations disease free

Germline - to correct the genetic disorder in the germ cells so that any children produced will have the corrected genes, not the defective genes.

4. Currently, it will be best to go for somatic treatment to improve Lee\'s quality of life.

5. Gene therapy can be hazardous as the viruses can provoke an immune response, the genes can be inserted at positions which affect other genes and the viruses can insert themselves near oncogenes, and the viral promoter can thus cause reading of the oncogenes.

Gene therapy is also expensive, has low efficiency and has safety and ethical issues.