Gene Therapy for CF

Question 1

What is the main obstacle with gene therapy for CF (or airways disease)

Answer 1

Working aginst the body’s defense mechanism

Clears out the genetic therapy vector as if it was a pathogen/particle

Question 2

What are two vecotrs used in gene therapy (in terms of delivering genetic material to target)

Answer 2

• Virus
• Liposome

Question 3

What is the advantage of using a viral vector in gene therapy

Answer 3

Successful delivery of gene and the target cells express the corrected protein

Question 4

What is the disadvantage of using a viral vector

Answer 4

Effect lasts for 2-3 weeks - can not be re-administered due to body’s adaptive immune response

Question 5

What is the advantage of using a liposome

Answer 5

Can be repeatedly administered without reduction in efficacy

Question 6

What is the disadvantage of using liposomes as vectors?

Answer 6

Not very effective compared to viral vectors

Wet lungs (flu-like symptoms)