Future treatments - gene therapy Flashcards
1
Q
What is gene therapy?
A
“The insertion of usually genetically altered genes into cells especially to replace defective genes in the treatment of genetic disorders or to provide a specialized disease-fighting function”
2
Q
What is genome editing?
A
” the use of biotechnological techniques to make changes to specific DNA sequences in the genome of a living organism”
3
Q
give examples of three genome editing tools
A
- zinc finger nucleases
- talen
- crispr/cas9
4
Q
Give example of viruses used to deliver DNA/RNA etc into cells
A
- adenoviruses
- adeno-associated viruses
- lentiviruses
- retroviruses
5
Q
What are the advantages/disadvantages with using adeno-associated viruses as vectors?
A
Advantages:
- do not cause any human diseases
- low immunogenicity and cytotoxicity
- limited integration to host genome
Disadvantages:
- low packaging capacity
6
Q
What are the main challenges of gene therapy
A
- cost
- immune response
- best route of administration?
- off target effects
- legislation and ethical implications
