Big news in the CF world
- lungwaytogo
- May 27, 2023
- 3 min read
Its been announced that early this year that the first stage of human trails. I've been researching these since my teens. For a fun little side but of research I highly recommend looking into early gene therapy on mice that looked at improving athletic performance. Mostly physical strength/ muscle aging (amusingly called Schwarzenegger mice) and cardio performance (a.k.a Marathon mice). This was the first study that seemed to show promise on gene therapy as a whole for people living with disabilities. I mean they also legit made a huge number of bunnies glow in the dark to test genetics through generations but that doesnt seem to help much unless you lived somewhere really dark. I often used to say I'm more than happy to be the first human guinea pig for the muscle and cardio genomes (as long as I don't get too big) if the only cost is I glow in the dark too. I'd just be really fun at parties 😅
Still I knew this was a long way off helping us find cures for disabilities. It was just a positive goal. They had a few set backs and some massive break throughs in the states from religious groups protesting "playing god" which in their defence we did amazing research the point we could effectively have "designer babies" after they did amazing work on in utero and gene therapy. Which in my opinion is too far. But should that lead to the point it could cure a baby before its even born I will support that wholeheartedly.
And more excitingly is this is not the transplant gene modification therapy that I have been campaigning for with the heading to 22. That's more for people in similar situations like me that may be able to harvest some stem cells or be viable for donated stem cells to be used to not only, effectively 3D print a non rejectable organ or tissue but also modify it to remove the faulty genome sequence that causes things like diabetes, cystic fibrosis and MS etc. This on the other hand is the first test way of the methods like CRISPR are being used on humans to treat a disability.
CRISPR has been viable for many years now however the real struggle has been finding a way to get it into the system of humans. The lesser effective of the methods was small enough to attach to a cold virus so it was able to reach the relevant cells on the back of that. But the more precise and effective CRISPR was much too large. In case I have this information incorrect or if im confusing anyone, give it a Google search.
This is really exciting to be honest. I know its a long long way off but for someone researching this for the last 16 years its like the end is finally in sight. Ive said it a lot on here about where my optimism and drive comes from and this is where it all started I guess.
Ive always had the idea that while I know my health will decline and some opportunities treatment wise will no longer be an option for me as the years pile on but I wanted to try and just say well enough to give myself the best chance of being viable for a possible treatment in the future. Be that keeping these lungs well enough for when the stem cell organ transplant comes available or when this gene therapy treatment sees clinical release. I know there's no guarantee so like with the release of Trikafta and Kaftrio I want to campaign for those in the future that may be a little more lucky than me or have fought a better fight.
If you want read more about gene therapies on the CF trust website I've posted the link below.
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