Gene therapy/treatment of genetic diseases - RNA treatments and techniques
3 important questions on Gene therapy/treatment of genetic diseases - RNA treatments and techniques
Modification of pseudogenes? (spinraza/ nusinersen)
- You creat form the pseudogene the nromale protein or you upregulate the speudogenes, so you can get the normal protein.
- induces the right splicing to rescue the pseudogenes and the phenotype
How happens the standard CRISPR-Cas9 method?
- It creates mostly knockouts but also gene editing is done with this type of CRISPR.
- Cas( is combined with a guideRNA and traceRNA (the RNA guid can bind close to the PAM code (NGG))
- Cas9 cuts both DNA strands in living cells
- Than you can have non-homologous end joining (leading to deletions/insertions making mostly aa knockout) or homology directed repair
(donor DNA/template DNA insertion)
- This CRISPR has off-targets effects.
CRISPR prime editing?
- Is a methode for specific editing of 1 nucleotide.
- The cas9nickase leas to a nickase cut, only the backbone is cut form one nucleotide.
- The guideRNA is now called the pegRNA (it is longer and complexer with two hairpins (bindingregions)).
- You can specificly choose which strand you want to cleavage
- precise gene editing
- dubble strande break and template is not nessacery.
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