Gene Therapy

Question 1

What is gene therapy?

Answer 1

• an experimental genetic engineering technique that replaces a faulty gene or adds a new gene
• in a n attempt to cure disease or improve the body’s ability to fight disease

Question 2

How are new genes introduced into the body?

Answer 2

• vectors such as viruses are used to deliver the gene into the cells of the body

Question 3

What are the types of delivery of new genes into the body?

Answer 3

1. Direct delivery
2. Cell-based delivery

Question 4

How does direct delivery of new genes work?

Answer 4

• the vector can be injected or given intravenously directly into a specific tissue in the body
• where it is taken up by individual cells

Question 5

How does cell-based delivery of new genes work?

Answer 5

• a sample of the patient’s cells can be removed and exposed to the vector in a laboratory setting
• the cells containing the vector are then returned to the patient
• if the treatment is successful, the new gene delivered by the vector will make a functioning protein

Question 6

What technical challenges must researchers overcome in before gene therapy becomes a practical approach to treating a disease?

Answer 6

• find better ways to deliver genes and target them to particular cells
• ensure that new genes are precisely controlled by the body