New company to use CRISPR/Cas9 to correct 80% of 3000 mutations involved in Duchenne muscular dystrophy

“The new company, based in Cambridge, Massachusetts, will use the CRISPR/Cas9 gene-editing technique known as SingleCut with the aim of correcting up to 80 percent of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD).”

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Good news, I just wish we would hear about something like this for schizo.

Not possible at this stage. No single known mutations are very important by themselves.

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I dont know why people on this forum keep saying stuff like that, We DO know quite well what the main culprit genes are.

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We do? This is new to me. When did we find these?
Some info would be much appreciated.

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There was a study at the broad and other unis a few years ago with thousands “i forget the exact number something like 40k” of schizophrenics they found about 108 genes that are affected and the main culprit was the C4 gene.

https://www.nature.com/articles/nature13595
Sorry its 108 loci so about 600 genes.

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Thank you for posting @twinklestars !
You are making an outstanding contribution to the news section of this forum.
This is a great piece of news for sufferers of Duchenne muscular dystrophy, and of course there is the potential
that CRISPR, or other gene modification techniques, will be used to treat other disorders as well,
and as well enhance the genetic parameters of healthy adults who want a boost.

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Thanks. I had a quick look at it. But if I’m understanding this correctly, the odds of developing schizophrenia are just estimated to be around 15 times higher if you have all 125 SNPs spanning these 108 locis.

Is this correct?

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Thanks treebeard for fixing my headline.

There are some genetics with a very high association with schizophrenia, like 22q11.2 deletion.

Most people don’t have that. They have many small genetic differences added together.

But perhaps in the future, multiple mutations can be corrected at one go.

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Sounds about right i dont care to read that article again right now.

Do we have enough knowledge about which genes interact to create the genetic risk for schizophrenia? I’ve been taught that even with our best estimates, we can’t use mutations to predict the risk for schizophrenia very well. But this might be outdated knowledge for all I know.

I think right now researchers can assign someone a genetic risk score (high or low) based on loading of risk genes. That is not something anyone in the public could get as far as I know - just in research.

I don’t know if they will be able to predict schizophrenia with genetic information only, but proteomics is advancing really fast too and that is looking likely for diagnosis and prediction.

This is one of those fields where large study populations and machine learning are necessary.

I’d say there will be major advancements within 5 years. There’s already a name for it, the “schizophrenia interactome.”

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Things are progressing so quickly when it comes to this. With most of my textbooks (not genetics or even mainly biology), I just skim these parts because I assume they’re pretty irrelevant, or will be soon enough. Which is pretty exciting, really. I hope they do make some breakthroughs now. They’ve been talking about the breakthroughs they’re about to make for decades, but that was before we had this kind of technology.

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Yeah it’s going to get kind of wild, I don’t know how doctors are going to keep up. Computer assisted medicine, I suppose.

And of course development of tests, treatment, medicines etc is always going to lag significantly behind the research.

Check this out though:

https://www.nature.com/articles/npjschz201612

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That’s really cool! It’s 12 AM here and I’m feeling my Seroquel now, but this looks amazing! So many new potential treatments and new ways to research the pathogenesis of sz. I love the gene-drug interactome.

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I’m just going to read my Merlin book until I fall asleep and try to convince myself that I don’t really want to change the field I’m studying to something more related to this. :roll_eyes: :mage:

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80% of 3000 mutations, that means correcting 2400 mutations… isnt sz less than that? How many mutations do sz supposedly have?

I think i didnt understand the article. I was under the impression that they were going to correct 80% of 3000 mutations in one go. As in doing 2400 crispr in one go.

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I didnt understand too… holy fu ck … it seems like they dont do anything crisper for sz… or it will take a longer time…!!