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What is gene therapy ?

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


What is somatic gene therapy ?

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.


What is germline gene therapy ?

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


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

A vector


What are the two main types of vectors ?

1. Attenuated or modified viruses
2. Non-Viral Vectors such as liposome transfection and Gene Gun


How does a gene gun work ?

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


What elements control gene expression?

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