1.2.13 CF treatment

Question 1

What are some of the current treatments for CF?

Answer 1

• Medication
• Diet
• Digestive Enzyme supplement
• Physiotherapy
• Heart and lung transplant

Question 2

What is gene therapy?

Answer 2

The genotype of targeted cells is replaced with healthy alleles

Question 3

How is gene therapy done using a virus?

Answer 3

• The DNA allowing the virus to replicate is removed
• This is replaced with a normal allele along with a promoter sequence
• Initiates transcription and translation
• The viral DNA is incorporated into the cell’s DNA

Question 4

How is gene therapy done using liposomes?

Answer 4

• A copy of the normal allele is inserted into a plasmid
• The plasmids then combine with liposimes (spherical phosolipid bilarers)
• The +ve phospholipid binds with the -ve DNA
  
  • Liposome-DNA complex is formed
• The patient breathes this in
• The liposomes fuse with the epithelial cells, carrying the DNA

Question 5

How often must gene therapy occur to treat CF?

Answer 5

Treatment must be regular as the DNA sequence is lost in replication

Question 6

Why is germ line therapy not permitted in the UK?

Answer 6

There are ethical objections to germ line therapy

Question 7

What type of gene therapy is allowed in the UK?

Answer 7

Somatic therapy is allowed in the UK