Genetic therapy for retinal disease
8 important questions on Genetic therapy for retinal disease
What are the 4 ways of genetic therapy?
- Adding DNA
- Fixing RNA
- Degrading bad RNA
- Fixing DNA
How does gene augmentation therapy work?
You have a DNA construct consisting of:
1. Promoter --> drives expression of cDNA
2. CDNA gene --> produces wild-type protein
3. Poly-a-tail --> needed for protein synthesis
What can a mutation in RPE65 cause?
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When can't gene augmentation not be applied for all the genes mutated in IRD (inherited retinal diseases)?
- regulating expression levels may be needed
- [gene] cDNA does possible not fit into AAV (adeno-associated viruses
What are antisense oligonucleotides (AONs)?
What are the pro's and con's of naked-AON and AAV-AONs? (Adeno-associated viruses, antisense oligonucleotides)
Pro's: Easy delivery, no permanent expression, adjusting dose possible
Cons: life long injections
AAV-AON
Pro's: single surgery, long term expression
Cons: side-effects persistent, partially reaching retina
What disease is a mutation in ABCA4 causing?
- normally involved in transport
- malfunctioning --> accumulation of toxic waste products (lipofuscin)
- affect pre-mRNA splicing --> fixed by AON therapy
What are the three genome editing strategies?
- Zinc finger nucleases --> proteins, less versatile
- Transcription activator-like effector nucleases (TALENs)
- CRISPR-Cas9
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