4 ms·

So Luxturna is a DNA sequence embedded in the AAV2 virus (https://en.wikipedia.org/wiki/Voretigene_neparvovec https://en.wikipedia.org/wiki/Voretigene_neparvove

So Luxturna is a DNA sequence embedded in the AAV2 virus (https://en.wikipedia.org/wiki/Voretigene_neparvovec https://en.wikipedia.org/wiki/Voretigene_neparvovec). It seems like they modified the Kozac (header, basically) of the DNA slightly (see Wikipedia). But essentially what they're doing is injecting the 'correct' DNA sequence into people with a biallelic (both copies) mutation on the RPE65 gene. The AAV2 virus is there as a delivery mechanism to get the DNA into the cells more efficiently. The injected DNA then hangs around (loose, I think) in the cell and will get read and the correct proteins get manufactured even though the patients main copies of the gene are faulty. The virus bit might be hard to do, and maybe editing the header of it. I'm not sure. I guess that probably is quite hard. But manufacturing DNA and injecting it is fairly trivial, as in this guy with a PhD and a small lab was able to do it for about $400. There are biotech companies who will just make the DNA for you. https://www.theguardian.com/science/2017/dec/24/josiah-zayner-diy-gene-editing-therapy-crispr-interview https://www.theguardian.com/science/2017/dec/24/josiah-zayne... ... this was using CRISPR which is like a DNA find/replace, so in some ways more sophisticated than the way Luxturna works. I'm not saying anything about the price of this treatment per se, obviously a load of R+D and regulatory ballet had to be done here, but just saying the basic building blocks of some gene therapies are quite a bit cheaper than I imagined.

zimpenfish 9y ago
Although the opinion of real biologists seems to be that Zayner is a snake oil peddling hack who doesn't know what he's doing. e.g. https://freethoughtblogs.com/pharyngula/2017/10/14/i-could-use-bigger-muscles-but-im-not-going-to-get-them-with-biotech-in-my-garage/ https://freethoughtblogs.com/pharyngula/2017/10/14/i-could-u...
- deleted 9y ago
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- codeulike 9y ago
  That guy misunderstands the point of Zayners stunt, and also misunderstands the kits that Zayner sells (they are kits for using crispr on yeast in a Petri dish). Zayner acknowledges that his arm injection stunt is likely very inefficient. Still I'm not going to defend Zayner, he probably is a bit of a dick. But the point is that you can modify the DNA of at least a small number of your own cells in a rough untargeted way for about $400. That's quite surprising. Makes you wonder what might be going on behind closed doors elsewhere. edit: Here's Zayner's write up of his stunt: http://www.ifyoudontknownowyaknow.com/2017/10/the-first-human-to-attempt-crispr-gene.html http://www.ifyoudontknownowyaknow.com/2017/10/the-first-huma... (possibly dodgy ads, adblock recommended)
Amygaz 9y ago
Without getting into details, gene editing has come a long way, and every steps involved are about 20-25% of the cost of what they used to be. Even FDA trials typically require lower enrollment and assays. So gene therapy should not be more expensive, it should be cheaper.

zimpenfish 9y ago

Although the opinion of real biologists seems to be that Zayner is a snake oil peddling hack who doesn't know what he's doing. e.g. https://freethoughtblogs.com/pharyngula/2017/10/14/i-could-use-bigger-muscles-but-im-not-going-to-get-them-with-biotech-in-my-garage/ https://freethoughtblogs.com/pharyngula/2017/10/14/i-could-u...

deleted 9y ago
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codeulike 9y ago
That guy misunderstands the point of Zayners stunt, and also misunderstands the kits that Zayner sells (they are kits for using crispr on yeast in a Petri dish). Zayner acknowledges that his arm injection stunt is likely very inefficient. Still I'm not going to defend Zayner, he probably is a bit of a dick. But the point is that you can modify the DNA of at least a small number of your own cells in a rough untargeted way for about $400. That's quite surprising. Makes you wonder what might be going on behind closed doors elsewhere. edit: Here's Zayner's write up of his stunt: http://www.ifyoudontknownowyaknow.com/2017/10/the-first-human-to-attempt-crispr-gene.html http://www.ifyoudontknownowyaknow.com/2017/10/the-first-huma... (possibly dodgy ads, adblock recommended)

Amygaz 9y ago

Without getting into details, gene editing has come a long way, and every steps involved are about 20-25% of the cost of what they used to be. Even FDA trials typically require lower enrollment and assays. So gene therapy should not be more expensive, it should be cheaper.