1. What is gene therapy? Do now: In your own words,
Define Genetic Condition
What do all genetic conditions have in common?

2. Do now: In your own words, Define Genetic Condition What do all genetic conditions have in common
Based on this, can you think of one way they could all potentially be treated or even cured?

3. Vocabulary
Vectors
Virus
Germline gene therapy

4. What is gene therapy?
Experimental technique that uses genes to treat or prevent disease.
Will allow doctors to treat a disorders by inserting genes into patient’s cells instead of using drugs or surgery.

5. There are several approaches to Gene Therapy
Replacing a mutated with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene
Introducing a new gene into the body to help fight a disease.

6. How could we introduce new genes into a person’s genome?

7. How does Gene therapy works?
A gene that is inserted directly into a cell usually does not function.
Instead, a carrier called a vector is genetically engineered to deliver the gene to the nucleus.
Viruses modified so they can’t cause disease are often used as vectors because they can deliver the new gene by infecting the cell.

8. How does it work?

9. Research needed There are many challenges in gene therapy.
Better ways to deliver genes and target them to particular cells are needed.
It also needs to be ensured that the new genes are precisely controlled by the body.

10. Is gene therapy safe?
Several studies have already shown that gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.
New techniques involve unpredictable risks.
Institutes involved to ensure safety of the clinical trial research patients include:
The National Institutes of Health (NIH)
The U.S. Food and Drug Administration (FDA)

11. Is Gene therapy available today?
Only in a research setting.
The U.S. Food and Drug Administration (FDA) has not yet approved any gene therapy products for sale in the United States.
Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS.

12. Current gene therapy research
Focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells.
Cannot be passed on to a person’s children.
If targeted to egg and sperm cells (germ cells) would allow the inserted gene to be passed on to future generations.
This approach is known as germline gene therapy.

13. Can you predict some Ethical issues concerning Gene therapy?

14. Ethical Issues in Gene therapy
Because gene therapy involves making changes to the body’s set of basic instructions, it raises many unique ethical concerns.
What are “good” and “bad” uses of gene therapy?
Will the it only be available to the wealthy?
Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?
Because people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment.
Could the widespread use of gene therapy make society less accepting of people who are different?
Who decides which traits are normal and which constitute a disability or disorder?

15. Video

16. Exit slip Write down 3 things you learned today .
Today I learned that…