John R. Hoffman is a Professor of Biology, public speaker and a scientist examining the recovery of the nervous system after injury. Since 2006 he has written several unpublished manuscripts and he is currently working on the first Nathaniel Smythe novel and short story collection. He spends his spare time with his family and running.

32 Somatic Gene Therapy

Objectives

By the end of this lecture, the student will be able to:

  1. Explain how gene therapy can be used for the treatment of a disease.
  2. Compare and contrast somatic and germ line gene therapy.

Part 1: Somatic Gene Therapy

The first half of this lecture on somatic gene therapy looks at how gene therapy is conducted in human patients. The handout for this presentation can be downloaded and printed.

Part 2: A brief history of Somatic Gene Therapy

The second half of this lecture on Somatic Gene Therapy looks at how gene therapy is regulated and a brief history of gene therapy. The handout for this presentation can be downloaded and printed.

Reading:

Sade RM & Kushf G (1998) Gene Therapy: Ethical and Social Issues. J So Carolina Med Assoc 94(9):406-410.

Gene Therapy. Genetics Home Reference.

Assignment.Somatic Gene Therapy: a “cure?”

In somatic gene therapy, a new or replacement gene is inserted into an adult’s body cells. The patient is able to make an informed decision about the risks and benefits of the treatment, and there is no risk of passing the altered genome to their children.

Within you discussion group, discuss the types of genetic conditions that should be treated with somatic gene therapy. Consider different types of situations including life-threatening conditions, unpleasant conditions, or aesthetic/cosmetic conditions.

Previous – 31 – Genetically Modified Organisms

Next – 33 – Germline gene therapy