Experimental Gene Therapy Use On Humans

1. Experimental Gene Therapy Use On Humans

2. What is gene therapy? • Gene therapy is a method of curing genetic disorders by introducing functioning genes into a patient’s body. • These functioning genes are supposed to correct and replace the defective genes responsible for genetic disorders. • There are two types of gene therapy. Somatic gene therapy and germ-line gene therapy.

3. Somatic gene therapy Vs. Germ-line gene therapy • Somatic gene therapy treats patients by introducing the genes to body cells like bone marrow or blood cells. • Germ-line gene therapy targets egg and sperm cells. It is medically impossible at this moment. This type of gene therapy would pass the inserted gene to future generations.

4. How does gene therapy work? • Genes enter the body of a patient through a carrier molecule called a vector. Vectors are usually viruses that have been modified so that they don’t harm the patient. • Common viruses used as vectors: retrovirus, adenovirus, adeno-associated virus, herpes simplex virus, and poxvirus. • Vectors are either injected or given through an IV. • There are nonviral ways of delivering genes.

5. Quick history lesson, SON! • Concept of gene therapy originated in the 60s and early 70s. • On September 1990, the first gene therapy procedure was approved. • In 1999, Jesse Gelsinger, a gene therapy clinical trial patient, died four days after beginning his treatment from multiple organ failures. • In 2008, the first gene therapy trial testing for inherited blindness was shown to improve sight.

6. Safety • Because of what happened in 1999, the FDA placed a temporary halt on all retroviral vectors used in blood stem cells. • FDA oversees and regulates all gene therapy products and research in the US. • The National Institutes of Health determine whether or not a clinical trial raises any ethical, safety, or medical concerns. • The Institutional Review Board and the Institutional Biosafety Committee must authorize each gene therapy clinical trial before it is put into action.

7. Ethical Issues • Germ-line gene therapy passes genes to future generations. Is it right to decide what genes should be passed on? • Is it right to use to germ-line gene therapy to improve human traits? • Is somatic gene therapy more or less ethical than germ-line gene therapy?

8. My viewpoint • Germ-line gene therapy should be hands off, with the exception of using it to get rid of genetic disorders that run in families. • Gene therapy should be used to treat serious diseases that have no other treatments.