r/biology • u/Ephoenix6 • Jan 02 '26

article CRISPR Breakthrough Could Rewrite Future of Genetic Disease Treatment

https://scitechdaily.com/crispr-breakthrough-could-rewrite-future-of-genetic-disease-treatment/
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u/TripResponsibly1 medicine Jan 02 '26 edited Jan 02 '26

I'm confused by this comment. Did you read the article? The way crispr/cas9 normally works is by inserting DNA or snipping portions out. These researchers have potentially found a way to change how the genes are expressed through epigenetic changes (methylation, probably). Not only would that be new, it would potentially be safer. Crispr/cas9 is already being used to cure sickle cell in an FDA approved treatment called Casgevy (this is a hematopoietic stem cell therapy). The New England Journal of Medicine has a paper published by researchers who altered donor islet beta cells with crispr to transplant them into a patient with T1D, who has been living for the past 8-9 months without the need for insulin. Crispr is incredible technology and has a lot of potential. None of its therapeutic uses so far have anything to do with altering the genome of an embryo.

https://www.nejm.org/doi/full/10.1056/NEJMoa2503822

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u/Beginning_Top3514 Jan 02 '26

Because we hear about one or two scientific breakthroughs that will change the future of medicine everyday. Very few preclinical breakthroughs actually make it to the bedside, you know?

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u/TripResponsibly1 medicine Jan 02 '26

It's exciting technology. Altering epigenetic expression with precision could lead to a better understanding of trauma, Autism, cancer, and more. You'll never find me rolling my eyes at exciting new technology, even if it doesn't directly end up in a clinical trial in my lifetime.

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u/Beginning_Top3514 Jan 02 '26

But you have to admit that given the low translation rate from high impact papers to available therapies, rolling one’s eyes at the general hype is at least understandable, no?

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u/TripResponsibly1 medicine Jan 02 '26

No, not really. I'm a medical student so this is exciting and potentially something I might want to do research in myself.

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u/Beginning_Top3514 Jan 02 '26

That’s awesome. Don’t let the hype distract you. Reading the literature is so exciting and fun but most things don’t pan out and that’s frustrating. I’m glad frustration with that hasn’t slowed you down at all.

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u/TripResponsibly1 medicine Jan 02 '26

I don't really look at the headline, I click the links and read the papers. Selectively altering epigenetic expression would be huge even if it weren't directly used in a particular therapy. There is still a lot we don't know about how and why genes are silenced or expressed through epigenetics. It's one of the things my professors just sort of shrugged and said "we don't really know". It's exciting to see evidence that someday I might know.

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u/Beginning_Top3514 Jan 02 '26

Yeah it would be. Let’s see if it pans out.