6.3.11 Gene Therapy

Question 1

What is gene therapy?

Answer 1

Altering a person’s genetic material to treat disaeses

Question 2

Note on gene therapy

Answer 2

• A scientists develop their understandings of the human genome
• The location of genes which causes genetic disorders can be found
• These faulty genes can be inactivated or replaced with a newly inserted gene

Question 3

How can vectors be used in gene therapy?

Answer 3

Viruses
- Can recognise cells and deliver genetic material into them

Non-Viral Vectors
- Liposomes and ‘naked’ DNA

Question 4

What are the 2 types of somatic gene therapy?

Answer 4

Ex Vivo
- New gene inserted via a virus vector into the cell
- BUT DONE OUTSIDE OF THE BODY
- Blood or bone marrow cells extracted and exposed to the virus which inserts a gene into the cells
- Cells are grown in a lab then returned to a person by injection into a vein
.
In Vivo
- New gene inserted by a vector into cells
- WHILE CELLS ARE INSIDE BODY

Question 5

What diseases can gene therapy treat?

Answer 5

• SCID
• Leukaemia
• Inherited Eye diseases

Question 6

Cons with Gene therapy

Answer 6

• Frequent injections are required
• Cells are replaced by the body and it is illegal to insert genes into humans which multiple and are passed down generations

Question 7

What is germline gene therapy

Answer 7

• Genetically modifying sperm or egg cells to create new offspring
• But illegal, changes made to genetical material in gametes or early embryos can potentially be permanent and could be inherited by future generations