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.

  • Getting back into the scene, looking at providing some new options, but will need to change some things and come back as a different later.

    Anyways, I can help with AAV as Grey mentioned. It's possible that with ~10,000 I can cure him/her. However it would be an entirely untested therapy and to make is more secure/longer lasting then it will require a potentially more dangerous therapy.

    Now this is a little harder because there is no way to delete a gene to fix things (easiest solution to a genetic based problem).

    What I would do is I would offer you the option of a few therapies, being likely longer lasting or shorter. I would then create the designed virus and insert the functional gene into your friends cells. We do this either outside of his/her genome (likely wont last as long) or directly insert into the genome (likely lasts longer).

    Now this will very likely slightly increase risks of cancer. We have no way of knowing exactly how severe that may be, but based on my plan and methods it would likely be rather quite low.

    If you want to talk more about this send me a message.

  • Realty Wizard.. where do I know you from? What have you done?
  • @RealityWizard said:
    Anyways, I can help with AAV as Grey mentioned. It's possible that with ~10,000 I can cure him/her. However it would be an entirely untested therapy and to make is more secure/longer lasting then it will require a potentially more dangerous therapy.

    ...

    What I would do is I would offer you the option of a few therapies, being likely longer lasting or shorter. I would then create the designed virus and insert the functional gene into your friends cells. We do this either outside of his/her genome (likely wont last as long) or directly insert into the genome (likely lasts longer).

    If we haven't managed to perfect genetic therapies to do a relatively harmless things like knock out myostatin in adult humans, even though plenty of millionaires would gladly throw their millions at such a thing at the slightest hope that they can stop going to the gym, I have serious doubts that you are sitting on a secret cure/treatment for cystic fibrosis that you are willing to part with for only 10K.

    We're all about self experimentation, but exploiting money from desperate people with little hope of success seems like something else entirely. Requesting that further conversation happen in private makes this seem even more predatory.

    You've been here for years, so I want to give you the benefit of the doubt here, but...

  • No, I have no true specials, I'm just willing to try something no one else is. I'm tired of seeing people give up because there might be some slight issue or because it might not work immediately or might be difficult. My plan would be to literally insert a new gene into this persons genome using a virus (likely AAV). As far as I know no one has actually tried this.

    The cost would also be literally exactly what I need to purchase the materials. I don't have enough money to do this for free (or really to even subsidize this), but I'm not in it to make money (well not from people in need, but if you want a myostatin KO to look better maybe I'll try for a profit).

    You mention millionaires wanting myostatin KO. I'm not so sure there are many willing to pay for it, not because they're not willing to pay, but because they don't know, don't care, or don't trust/believe in possible results.

    I mostly want to think about this and consider all the options of therapy. Like I said I give no guarantee, but I believe that it will work and dearly hope it will, because whether or not I do it someone will and someone might just solve this issue.

Sign In or Register to comment.