CRISPR gene therapy

[jazzboypro]

CRISPR gene therapy seems to cure dangerous inflammatory condition

Nine out of 10 people who received a new version of a CRISPR-based treatment for a potentially life-threatening inflammatory condition seem to have been cured.

https://www.newscientist.com/article/2415156-crispr-gene-therapy-seems-to-cure-dangerous-inflammatory-condition/#:~:text=Nine people with a rare,a CRISPR-based gene therapy.

[NSXCIGAR]

I've been hearing about this CRISPR for years. Was wondering what was going on. 

Kind of the same thing with graphene and quantum computing. Let's get this stuff moving. 

[jazzboypro]

I agree, i know the FDA approved a CRISPR treatment for people with sickle cell disease. Apparently it worked well. It's still too expensive to be a mainstream treatment but it looks like it will be a huge thing in the coming years. 

[Diabolicalpherpher]

It's a very new and effective technology in molecular biology and genetics.  I've dabbled in CRISPR/CAS9 gene editing and what I know is different from what people think it can do.

You cannot change your entire DNA in every cell. So it is not an end all cure. 

This technology probably works well in genetic mutations in blood cells where you can kill off all the bad blood cells in you  including the stem cells with the disease mutation and introduce 'engineered stem cells' from your own cell that has been 'cured' and injected back into the body.

So essentially you get your skin cells turned into iPS cell and then use CRISPR/Cas9 to make your mutation back to normal and then force the corrected iPS cell into a certain lineage and then inject it back into the person.

This is very much an individualized medicine and the fact that you have to do all of those things in the  clinical lab to generate the cells make it very expensive.

Because you don't change your own DNA in the germ cells (aka your sperm or egg) your offspring may still be affected or carry the mutation.

People think you can just inject something and change the DNA in every cell but it doesn't work like that.  

This technology only works to change a mutation back to the original and be able to be put back into you. The idea that this technology can create super human or organism is fiction because you don't know what genetic material to mutate to to get what you want phenotypically.

[jazzboypro]

6 hours ago, Diabolicalpherpher said:

It's a very new and effective technology in molecular biology and genetics.  I've dabbled in CRISPR/CAS9 gene editing and what I know is different from what people think it can do.

You cannot change your entire DNA in every cell. So it is not an end all cure. 

This technology probably works well in genetic mutations in blood cells where you can kill off all the bad blood cells in you  including the stem cells with the disease mutation and introduce 'engineered stem cells' from your own cell that has been 'cured' and injected back into the body.

So essentially you get your skin cells turned into iPS cell and then use CRISPR/Cas9 to make your mutation back to normal and then force the corrected iPS cell into a certain lineage and then inject it back into the person.

This is very much an individualized medicine and the fact that you have to do all of those things in the  clinical lab to generate the cells make it very expensive.

Because you don't change your own DNA in the germ cells (aka your sperm or egg) your offspring may still be affected or carry the mutation.

People think you can just inject something and change the DNA in every cell but it doesn't work like that.  

This technology only works to change a mutation back to the original and be able to be put back into you. The idea that this technology can create super human or organism is fiction because you don't know what genetic material to mutate to to get what you want phenotypically.

Very interesting. I know it's not a cure all solution but I'm willing to bet that in 10-20 years from now it will be able to do much more. I know they are currently investigation the possibility to cure certain type of blindness (they have successfully done it with mice). This is definitely cutting edge (no pun intended), I will be following the development of this.