1. Topic F 3
Gene Therapy

2. F.3.3 Two Major Types of Gene Therapy-
Somatic Gene Therapy – For just the patient
Germ Line Gene Therapy – For the patient and all of his/her progeny
Leber's congenital amaurosis
X-linked severe combined immunodeficiency

3. Bacterial Vectors
F.3.1
Reverse Transcriptase is the enzyme carried inside RNA viruses.
How does it aid in infection?
How is Reverse Transcriptase used? In gene therapy?
F.3.2

4. F.3.4
Use of Viral Vectors in Gene Therapy

5. F.3.5 Ethical considerations to make for Gene Therapy
TOK: There have been some recent cases in
countries around the world where subjects have died
as a consequence of participating in a gene therapy
research protocol. These cases could be examined to
consider such issues as safety, conflicts of interest and
other violations of ethical practice in research.
Review this Case Study and discuss the following considerations:
How safe must an experiment be before it is ethical to try it on humans?
Is the review process efficient?
Would you consider putting your own child in a gene therapy trial?
What if Jesse had the fatal form of ornithine transcarbamylase (OTC) ? Would your opinion change?