AgitatedHiatusI have a genetic disease called HCM (it's also hereditary). It'd be cool to have it fixed.
That's the other problem. You cant just go about changing stuff in people and expect it to work. If you have a genetic condition that causes developmental issues then you'd need to tackle those before they develop i.e. when you're like 8 cells large. Otherwise you need to hope your issue is in a tissue that has rapidly dividing stem cells like intestines.
If by HCM you mean the heart issue, then according to wikipedia, mutants of sarcomere (things that makes up muscles) proteins are to blame. Hypertrophy is probably because since muscles don't function as well, you need more of it to get normal cardiac output. I don't know if fixing those proteins would allow the heart to shrink, maybe it would. It's certainly worth a shot in mice.
Here's the real utility of crispr
, you could have your genome sequenced, find out the exact mutation you have and make the same exact mutation in some mice, let those mice develop from embryos, then attempt to use crispr and provide a repair template and fix the gene at various stages of life and see what happens. your biggest problem is delivering a crispr system and template to the heart. I guess a virus is the way to go but i'm not sure on that. That actually sounds pretty exciting I hope someone tries it.
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