Gene Therapy Flashcards
What is gene therapy?
The introduction of a specific gene into a patient as a therapeutic agent
When does using gene therapy to introduce a functional copy of a mutant gene work well?
For single gene disorders where a specific mutation in a specific gene is causing all the symptoms
Which diseases can be treated with the introduction of a functional gene copy?
Cystic fibrosis, metabolic disorders, immunodeficiency
How can the introduction of an inhibitory transgene be used as gene therapy?
When the overexpression of a product is what is causing disease
What is typically introduced as an inhibitory transgene for gene therapy?
A gene encoding miRNA for the faulty gene to knockdown its expression
What diseases are inhibitory transgenes used to treat?
Cancer, infections, some inherited genetic diseases
How do we get the gene into the cell for gene therapy?
Use modified viruses or viral-like vectors
Why do we use viruses to deliver gene therapy into human cells?
They have evolved to deliver genetic material into human cells, so are very good at it
What are 3 problems we have had with the viral vectors?
- Cause an immune response in the patients
- Size
- Genome integration
Why do we have problems with the size of the viral vector?
A large vector can carry more DNA, but is also more likely to cause an immune response
Why is genome integration a good thing with the viral vectors?
Genome integration will allow the inserted gene to be copied and passed on when the cell divides
Why is genome integration potentially a problem with the viral vectors?
The gene is integrated at random in the genome, so it could potentially end up in another gene and disrupt its function, leading to cancer
What are the two ways to deliver gene therapy?
In vivo and ex vivo
What does in vivo delivery of gene therapy involve?
The vector containing the transgene is injected directly into the patient
What are the challenges with in vivo gene therapy?
Hard to get the vector to target the correct tissue
