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Another Incredible CRISPR Gene Editing Breakthrough

We are just at the beginning of the CRISPR era, using the gene editing tool to eradicate a variety of diseases. Its potential is breathtaking. We first wrote about CRISPR in 2018:

Illuminate [2018]: "CRISPR” (pronounced “crisper”) stands for Clustered Regularly Interspaced Short Palindromic Repeats, and researchers have developed a new version, CRISPR-Cas9, to edit the base pairs of genes. Gene editing will change our world in ways we can't even imagine yet, and its already started. Mike Mcrae has a piece in Science Alert about ways scientists are adopting CRISPR technology in medicine, agriculture and the environmental sciences. And after successful tests in monkeys, researchers will begin using the gene editing tool on humans sometime this year to treat blood disorders like sickle-cell disease and beta thalassemia."

Since then, CRISPR has been successfully employed to battle a variety of illnesses.

NPR: "CRISPR has already been shown to help patients suffering from the devastating blood disorders sickle cell disease and beta thalassemia. And doctors are trying to use it to treat cancer and to restore vision to people blinded by a rare genetic disorder."

And, this week researched announced that it had been used to stop another deadly inherited disease known as transthyretin amyloidosis which results from the build-up of a misfolded transthyretin (TTR) protein, destroying important tissues such as nerves in his hands and feet and his heart.

On Saturday, researchers reported in the New England Journal of Medicine that the experimental treatment worked, causing levels of the destructive TTR protein to plummet.

New England Journal of Medicine: "Preclinical studies showed durable knockout of TTR after a single dose. Serial assessments of safety during the first 28 days after infusion in patients revealed few adverse events, and those that did occur were mild in grade."

NPR interviewed one of the test subjects, Patrick Doherty of County Donegal in Ireland, and he was ecstatic regarding the results saying, "I feel fantastic. It's just phenomenal."

The major breakthrough in this case is how the CRISPR gene editing tools were employed.

NPR: "Doctors infused billions of microscopic structures known as nanoparticles carrying genetic instructions for the CRISPR gene-editor into four patients in London and two in New Zealand. The nanoparticles were absorbed by their livers, where they unleashed armies of CRISPR gene-editors. The CRISPR editor honed in on the target gene in the liver and sliced it, disabling production of the destructive protein."

The technique shows that you don't have to inject CRISPR-Cas9 directly into specific cells; that you can simply inject them into the bloodstream to make edits, opening the door to use gene editing for the treatment of many common diseases.

#health #news #research

By: Don Lam & Curated Content

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