Gene Therapy Flashcards
What is gene therapy?
Is the use of genetic material to treat or prevent disease.
What is an example of gene therapy?
The human Genome project.
What does it mean for gene therapy to be used ‘in Vivo?
‘In Vivo refers to the gene being directly inserted in the person.
What does it mean for gene therapy to be used in ‘Ex Vivo?
‘Ex Vivo’ refers to cells from the patient being removed, genes being inserted into an altered virus, and then being mixed with cells from the patient to cause them to become genetically altered. These altered cells are injected back into the patient to then produce the desired protein or hormone.
What are 4 possibilities gene therapy can be used for?
- Replacing a mutated gene with a healthy copy.
- Fixing or inactivating mutated genes.
- Insert a new gene that will fight the disease.
- Making the immune system recognise diseased cells.
What diseases/conditions can gene therapy be used against?
- Cystic fibrosis
- Huntington’s Disease
- Type 1 diabetes
What is Type 1 diabetes?
Is an autoimmune disease where T cells attack beta cells causing a lack of insulin.
Describe how type 1 diabetes can affect the control of blood glucose levels.
Beta cells in the pancreas produce little insulin. Glucose is not taken into cells and is not able to be stored as glycogen, therefore blood glucose levels remain high.
How can gene therapy combat the effects of type 1 diabetes?
By reprogramming alpha cells to beta cells.
Describe how gene therapy can reprogramme alpha cells to beta cells to combat type 1 diabetes.
- Viruses containing proteins are inserted into the pancreas.
- These proteins change gene expression and cause alpha cells to produce insulin.
- These new beta cells are resistant to attacks by T cells as they are slightly different to regular ones.
