Module 13: Gene Therapy

Question 1

Somatic Gene Therapy

Answer 1

• Replacement of missing gene product by inserting normal gene
• Commonly used for loss of function mutations because of absent protein production

Question 2

Germline Gene Therapy

Answer 2

• Editing of genes in the eggs or sperm

- Impact of future generations

Question 3

Gene Transfer

Answer 3

• A vector (usually a virus)
• Gene to be delivered is placed inside the inactive virus
• A relevant target cell to deliver the RNA or DNA used to take gene to destination

Question 4

Viral Vectors

Answer 4

• Retroviruses
• Adenoviruses
• Adeno-Associated viruses
• Hermes simplex viruses
• Lentviruses

Question 5

Risks of Gene Therapy

Answer 5

• Retroviruses: integration into a portion of the genome that activates a proto-oncogene
• Adenoviruses: can cause an immune response

Question 6

What type of mutation is somatic gene therapy most successful in correcting?

Answer 6

Loss of function

Question 7

What 3 items are needed for successful somatic gene therapy?

Answer 7

Vector, isolated normal copy of the gene, & target cells

Question 8

Which of the following is NOT a consideration when selecting a viral vector?

Answer 8

Ability of virus to infect other species

Question 9

What mechanisms can induce tumor formation as a result of gene therapy?

Answer 9

Vector inserts next to proto-oncogene and turns it into an oncogene

Question 10

Why does gene therapy utilize adeno-associated viruses have to be re-administered?

Answer 10

The vector does not insert the corrected gene into the host cell

Question 11

What makes liposome gene therapy different from other gene therapy methods we have discussed?

Answer 11

It does not require a virus

Question 12

(T/F) Somatic gene therapy impacts the genome of the offspring of the recipient

Answer 12

False