3 ms·
Just an FYI - The R+D and govt approval process is expensive. The actual treatments are - I'd wager - relatively easy to produce. So this Novartis treatment is
by codeulike 7y ago
Just an FYI - The R+D and govt approval process is expensive. The actual treatments are - I'd wager - relatively easy to produce. So this Novartis treatment is delivering DNA fragments via AAV. Its not attempting CRISPR or anything like that, its just gets extra copies of the unmutated gene into the nuclei so the right proteins can get made. I think they call that upregulation. So its basically AAV plus DNA fragments.
So for example you can buy engineered AAV viruses online. They sell this stuff for research. Looks like its about $350 a pop
https://www.addgene.org/viral-service/aav-prep/ https://www.addgene.org/viral-service/aav-prep/
Novartis aren't using CRISPR here but as another example these guys sell CRISPR for your research experiments:
https://www.addgene.org/crispr/guide/ https://www.addgene.org/crispr/guide/
Here's some Crispr for plant experiments for about $400
https://www.addgene.org/kits/stuttmann-dicot-genome-editing/ https://www.addgene.org/kits/stuttmann-dicot-genome-editing/
So I'm sure there a lot more to it than that, but even so, it seems to me that the nuts and bolts of this stuff - once known - are going to be easy to produce. How long till we get a generic?