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The first CRISPR gene-editing drug is coming—possibly as soon as next year
Fast Company via yahoo ^ | 7/1/2022 | Sy Mukherjee

Posted on 07/05/2022 7:13:30 PM PDT by RomanSoldier19

ntil recently, CRISPR—the gene-editing technology that won scientists Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in chemistry—sounded more like science fiction than medicine; lab-created molecular scissors are used to snip out problematic DNA sections in a patient’s cells to cure them of disease. But soon we could see regulators approve the very first treatment using this gene-editing technology in an effort to combat rare inherited blood disorders that affect millions across the globe.

In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed promising evidence that it can help patients with beta thalassemia and sickle cell disease (SCD), both of which are genetic blood diseases that are relatively rare in the U.S. but somewhat more common inherited conditions globally.

Beta thalassemia is characterized by damaged or missing genes that cause the body to produce less hemoglobin (an essential protein that transports oxygen), potentially leading to enlargement of the liver, spleen, or heart, and malformed or brittle bones. It is estimated to afflict 1 in 100,000 people in the world, and regular blood transfusions are necessary to stave off its most serious effects.

(Excerpt) Read more at msn.com ...


TOPICS: News/Current Events
KEYWORDS: crispr; editing; gene

1 posted on 07/05/2022 7:13:30 PM PDT by RomanSoldier19
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To: RomanSoldier19

..binds to the genes that inhibit melanin production, and removes the protein syntheses pathway for ADP ....

It’s coming


2 posted on 07/05/2022 7:17:41 PM PDT by Technocrat
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To: RomanSoldier19

They call this the “Ezekiel Emanuel” drug. After taking the drug you live a healthy life until age 65/66 (depending on your country’s Social Security). You the die immediately!


3 posted on 07/05/2022 7:20:29 PM PDT by Jan_Sobieski (Sanctification)
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To: RomanSoldier19
>>>possible as soon as next year

Guess they never heard of the Experimental Gene Therapy China Virus shot.

4 posted on 07/05/2022 7:27:34 PM PDT by Deaf Smith (When a Texan takes his chances, chances will be taken that's for sure.)
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To: RomanSoldier19
Dang those pesky problematic DNA sections....
Dang them to Heck!


5 posted on 07/05/2022 7:38:48 PM PDT by GaltAdonis
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To: RomanSoldier19

The exa-cel gene editing process relies on ex vivo processing which requires taking hematopoeic (blood cell producing) stem cells from the patient, editing them to correct the gene defect, conditioning the patient’s blood with chemotherapy like drugs to kill off as many defective stem cells as possible without causing anemia or immune system problems, and then reinjecting the corrected stem cells back into the same patient so they can produce normal red cells. This is a first step that is very expensive and takes several months to complete making it available to a fairly limited number of candidates.

The real future of this therapy will be when CRISPR can taking already normal stem cells from healthy donors and knock out the genes that would trigger an immune response in future recipients (allogeneic). The generic stem cells can then be manufactured and ready to be injected into patients off-the-shelf with no delay. They also are working to condition the patients with gene therapies that naturally kill off just the defective stem cells without chemotherapy drugs making it far less risky to treat a wide range of patients. This will reduce cost and treatment time significantly.

CRISPR is doing similar trials with the islet cells in the pancreas which will allow them to treat Type I and II diabetes with a onetime cure and with allogeneic T-cells that carry cancer killing drugs to only the cancer cells while leaving health cells unharmed.


6 posted on 07/05/2022 7:46:13 PM PDT by Dave Wright
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To: RomanSoldier19

2019 - “there’s no way these people will accept genetic modification of their DNA”
2020 - COVID
2021 - vaccine
2022 - “guess what? Youve already had your DNA modified through reverse encryption. Now try our crispr drug!”


7 posted on 07/05/2022 7:51:41 PM PDT by struggle
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To: Technocrat

Please state exactly what is coming.


8 posted on 07/05/2022 8:07:14 PM PDT by Arcadian Empire (The Baric-Daszak-Fauci spike protein, by itself, is deadly.)
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To: RomanSoldier19

Releasing a gene piranha into the wild is problematic.


9 posted on 07/05/2022 8:08:26 PM PDT by Arcadian Empire (The Baric-Daszak-Fauci spike protein, by itself, is deadly.)
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To: RomanSoldier19

I have this but I am considered a carrier because I don’t have the type that requires blood transfusions. It’s hogwash when they say a carrier doesn’t have symptoms. My blood work looks disasterous and of course it causes issues...and you have to watch for quacks who tell you that you are anemic and need to take iron.
My G6pd is also messed up but no Dr knows enough to understand why. All they know about is G6pd deficiency


10 posted on 07/05/2022 8:17:17 PM PDT by RummyChick
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To: RomanSoldier19
They've been doing CRISPR Cas9 gene editing on food plants for a while. Gene edited lettuce is already in grocery stores.

This is a gene edited tomato plant they've been working on.

They figured out how to get rid of the main stem so they can grow them in indoor vertical farming where they already grow the lettuce. None of the plants will ever see sun. It's all LED lighting and robotics handles quite a bit of the labor.

No telling what all this food grown without soil or in sterile soil with NPK and micro-nutrients added will do to people's gut flora or immunity system. No bacteria, no fungi.

11 posted on 07/05/2022 8:39:28 PM PDT by Pollard (If there's a question mark in the headline, the answer should always be No.)
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To: Arcadian Empire

12 posted on 07/05/2022 9:13:20 PM PDT by RomanSoldier19 (Res ad Triarios venit)
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To: Arcadian Empire

China will create a transmissible virus which fatally modifies the DNA of all Caucasians and Negroids.


13 posted on 07/05/2022 9:45:22 PM PDT by Technocrat
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