Chapter 17.4: Human Gene Therapy

Question 1

Define in vivo gene therapy

Answer 1

genes are delivered directly to somatic cells

Question 2

What is ex vivo gene therapy?

Answer 2

 Ex vivo gene therapy is when researches remove tissues from the patient’s bone marrow, expose the cells to large amounts of the therapy gene while the cells grow briefly in culture, and then return the cells to the patient’s body

Question 3

What is a gene whose expression will fight disease?

Answer 3

therapeutic gene

Question 4

What is the main problem with retroviral vectors?

Answer 4

• One problem with retroviral vectors is that their integration can result in genome mutation and in some cases, can cause cancer in the patient

Question 5

What are the two type of viral vector involved in gene therapy trials?

Answer 5

retroviral vectors or adeno-associated viral vectors (AAV vectors)

Question 6

Define therapeutic gene

Answer 6

a gene whose expression will fight disease

Question 7

What means genes are delivered directly to somatic cells?

Answer 7

in vivo gene therapy