Therapeutic gene editing Flashcards
1
Q
List the advantages and disadvantages of CRISPR/Cas9 compared with TALENs and ZFNs
A
Advantages
- Utilises classic base pairing rules (compared with protein:DNA interactions)
- Easier, cheaper and quicker
Disadvantages
- Recognition sequence is relatively small (~20bp) compared with TALENs (30-40bp) and ZFNs (18-36bp)
- Increases potential off-target effects
2
Q
LIST the potential uses of genome editing
A
- Creating cell and animal models
- Screening to identify new therapeutic targets
- Gene drives
- Agriculture research
- Treating diseases
3
Q
How can gene therapy aid those who have HIV?
A
- CCR5 is critical protein involved in the binding and entry of HIV
- Found that those with 32 aa loss in CCR5 are resistant to HIV-1 infection
- Gene therapy using ZFN targets CCR5 gene in T cells and renders them permanently dysfunctional
