Friend fading with cystic fibrosis

Hi, new here and very much aware crispr is in very early stages but will become the future.

Where does the other tools/materials required come from or knowledge on how to synthesise.

Along with a kit, would any outside chance of experiment as a last resort be possible.

Aware of risks etc,

Also the drugs to treat have revently been made available to our health service but being denied access to them, there was even a mention of the wrong thing was sent, using this drug symkevi has only revently been passed here.

Thanks guys for any advice. Good or bad appreciated

Comments

  • How much money are you willing to spend on this, and what kind of time frame do you need stuff done by?

    As far as medications go, the 2 drugs I see, Orkambi and Kalydeco are available from some online pharmacies, however, they cost $15,000 USD and $24,000 USD respectively for 28 capsules. Unless you're a billionaire, I don't see that being an option.

    So, you can try immigrating to a country that has the drugs you need available. There are coupons for this sort of thing in America.

    As far as radical, bleeding-edge therapies go, my first thought is using some sort of retrovirus or AAV as a gene vector, but if I understand the way modern gene therapy works, this is a temporary solution, as the cells will eventually die off and new ones carrying the old genes will pop up again. Idk how crispr would even be applied here, since the target is cells already in the body.

  • I misread. The Kalydeco is 24k for 60 tablets.

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