5 ms·
I'm thinking about genetic diseases occurring in non-dividing cells like cortical neurons as a possibility. It seems like as long as the technology relies on NH
by heyoni 3y ago
I'm thinking about genetic diseases occurring in non-dividing cells like cortical neurons as a possibility. It seems like as long as the technology relies on NHEJ or HDR this kind of editing won't be possible as a therapy.
- dillydogg 3y agoNHEJ works in non-dividing cells, though HR requires it. Many of these techniques (prime editing in particular) use exogenous enzymes to mediate some of the repair and can work in non-dividing cells.
- heyoni 3y agoI must have misread some literature so thanks for clearing that up. I'm now seeing that it only requires a homologous template which, hypothetically could even be provided by the therapeutic agent itself?
- dillydogg 3y agoYes, this is actually how prime editing works! You also attach some reverse transcriptase to perform the base insertion (but the repair requires cellular help, prime editing works in non-dividing cells though!)
- heyoni 3y agoIs prime editing possible for diseases that result from point mutations? I know tat there’s always the issue of getting the construct into the cells and then into the nucleus but can we confidently elicit base edits in humans? All I’ve seen (and I haven’t seen everything) is crispr mediated transcription in Duchene muscle dystrophy but it failed. I’m looking at possible cures for satb2 but was under the impression that NHEJ wasn’t viable for some reason (my notes suck).