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CRISPR to halt the progress of Duchenne muscular dystrophy (DMD) in dogs. Potentially in humans, too

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Quest

 

https://www.digitaltrends.com/cool-tech/crispr-gene-editing-dmd/amp/

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CRISPR gene editing can be used for all manner of applications, from creating more efficient crops or heat-resistant cattle to coding GIFS into DNA. Now, scientists from the University of Texas Southwestern have showcased another potentially transformative use case for the technology: Using CRISPR to halt the progress of Duchenne muscular dystrophy (DMD) in dogs. One day, it could potentially do the same in humans, too.

 

DMD is the most common fatal genetic disease in children. The result of a mutation that limits the production of muscle-function protein dystrophin...Children who suffer with DMD often die as a result of their hearts lacking enough strength to pump, or their diaphragm being too weak to breathe...

In their demonstration, the UT Southwestern scientists were able to use a single-cut gene-editing technique to restore the dystrophin in muscle and heart tissue by a massive 92 percent. According to experts, a 15 percent threshold could be enough to significantly help patients.

Previously, the researchers showed that it was possible to correct DMD mutations in both mice and human cells. However, in this latest breakthrough, they demonstrated that it is possible to employ this strategy in dogs, by far the largest mammals this has been applied to. Four dogs were involved in the study, all of which shared the same genetic mutation seen in DMD patients. The CRISPR gene-editing components were delivered through a harmless virus, after which it proceeded to carry out the successful gene-editing work.

 

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Nietzsche

Great to see progress in CRISPR research is there any link to HSV

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dont quit!17
5 hours ago, Quest said:

I love anything positive! I need it succeed in any way! I want it to be around for us!

Here's Why CRISPR Stocks Rose as Much as 18.7% in August

https://www.fool.com/amp/investing/2018/09/04/heres-why-crispr-stocks-rose-as-much-as-187-in-aug.aspx

Thats good news. I've had young patients with Muscular Dystrophy and it is very debilitating genetic disease. They would probably trade places with most us here on this forum in a heartbeat. Good share. 

(some perspective to start my day)

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Quest
8 hours ago, dont quit!17 said:

Thats good news. I've had young patients with Muscular Dystrophy and it is very debilitating genetic disease. They would probably trade places with most us here on this forum in a heartbeat. Good share. 

(some perspective to start my day)

It sure does put it in perspective! I am not sure I would have worked on my health as much if I didn't know that I had a failing immune system! I really don't get colds so I would have thought my immune system was okay. I hope your patients get to benefit from it one day!

Edited by Quest

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Voyager2

Good find. This thing works. They used CRISPR to reduce muscular dystrophy by 92% in 4 dogs.

Excision Bio says their own CRISPR/HSV program will be in the clinic by 2021 (a phase 1 trial is 3 years away). Let's hope things move quickly at that point due to the increasingly widespread use of CRISPR around the world and lessons learned.       

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Quest

Everybody can learn from each other! I hope they do share their information not like those other scientists!

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Voyager2

Very good news! We've heard about unintended off-target cuts for awhile and wondered if it would delay the use of CRISPR. This article describes a logical way around the problem and with any luck it might not be an issue when Excision Bio starts HSV human trials in 3 years. Maybe they'll start sooner if they can routinely cure monkeys? 

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infoguy123

Ya it's awesome news. I can't wait hear that they've(functionally/therapeutically) cured 20 people in a trial 3 years from now. It will be almost as good as the actual cure because we'll know that it's just around the corner

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dont quit!17
8 hours ago, Voyager2 said:

Very good news! We've heard about unintended off-target cuts for awhile and wondered if it would delay the use of CRISPR. This article describes a logical way around the problem and with any luck it might not be an issue when Excision Bio starts HSV human trials in 3 years. Maybe they'll start sooner if they can routinely cure monkeys? 

You think Excision would pay attention to studies like these?

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35hope
18 hours ago, infoguy123 said:

Ya it's awesome news. I can't wait hear that they've(functionally/therapeutically) cured 20 people in a trial 3 years from now. It will be almost as good as the actual cure because we'll know that it's just around the corner

i know i wish some people from the companies would just bootleg the vaccines if they had some insider info on it being successful 

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Voyager2
On 9/15/2018 at 8:54 AM, dont quit!17 said:

You think Excision would pay attention to studies like these?

Excision needs to get around the off-targeting problem and are likely keeping an eye on possible solutions in the literature.

  

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Onehotmess

Some more good news on crispr!

Restoring cells to an uninfected state once a virus is destroyed.

https://phys.org/news/2018-09-cells-uninfected-state-virus.amp

Now if only they could remove this virus from each of us and fix the effective cells would be no longer a dream! 

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Divon
12 minutes ago, Onehotmess said:

Thats an article from an investors perspective, stock market to be exact. I would say that it is unlikely that researchers will not find any real use of CRISPR because it is so hyped to investigate possibilities with it atm.. However, people who invest hard on it do so with the hope that this will be the big thing in medicine that will cure everyone from everything and make old people young again. 

I have been saying again and again on this forum that you can´t put all your hope on CRISPR. There are a lot of other things to have hope for in the herpes treatment. CRISPR is obviously more exciting though because it might have a chance to kill the latent virus one day, however that day is quite far away by looks of it. 

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