What is gene therapy?Do now: In your own words,

Define Genetic ConditionWhat do all genetic conditions have in

common?

Do now: In your own words, Define Genetic ConditionWhat 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?

VocabularyVectorsVirusGermline gene therapy

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.

There are several approaches to Gene TherapyReplacing a mutated with a healthy copy of

the gene.Inactivating, or “knocking out,” a mutated

geneIntroducing a new gene into the body to help

fight a disease.

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

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.

How does it work?

Research neededThere 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.

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)

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.

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.

Can you predict some Ethical issues concerning Gene therapy?

Ethical Issues in Gene therapyBecause 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?

Videohttps://

www.youtube.com/watch?v=bLI1Gfb0ynw

Exit slipWrite down 3 things you learned today .

1. Today I learned that…2. Today I learned that…3. Today I learned that…