Gene Therapy - Whitt

Question 1

What is gene therapy ?

Answer 1

Gene therapy is a novel approach to treating diseases based on modifying the expression of a person’s genes toward a therapeutic goal.

Question 2

What is somatic gene therapy ?

Answer 2

Manipulation of the genes of a patient in a manner that provide the patient with benefit but will not be passed down to their offspring.

Question 3

What is germline gene therapy ?

Answer 3

Manipulation of a patients germ line genes in a manner that they will be passed to the next generation.

Question 4

How do you deliver your gene of interest to the target area in the genome

Answer 4

A vector

Question 5

What are the two main types of vectors ?

Answer 5

1. Attenuated or modified viruses

2. Non-Viral Vectors such as liposome transfection and Gene Gun

Question 6

How does a gene gun work ?

Answer 6

DNA can be conjugated to gold particles and delivered. Used primariy to give DNA vaccines

Question 7

What elements control gene expression?

Answer 7

You need a way to mantain the level of therapeutic peptide at the right time

It is also essential to maintain long term expression of genes in the correct tissues