CRISPR scores what looks like a win

[Rhea]

This sounds like a dramatic advance in the science. Injecting the CRISPR directly into the patient to reach remote problems, instead of drawing out a sample, editing and returing the sample.




https://www.npr.org/sections/health...a-crispr-gene-editing-breakthrough-stopped-it



The implications for Alzheimers and Parkinsons is huge, and for other devastating diseases.

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.

But those experiments involve taking cells out of the body, editing them in the lab, and infusing them back in or injecting CRISPR directly into cells that need fixing.

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The study Doherty volunteered for is the first in which doctors are simply infusing the gene-editor directly into patients and letting it find its own way to the right gene in the right cells. In this case, it's cells in the liver making the destructive protein.

I definitely feel better," he told NPR. "I'm speaking to you from upstairs in our house. I climbed stairs to get up here. I would have been feeling breathless. I'm thrilled."

 

[J842P]

Awesome.

 

[Tharmas]

Amazing. I too suffer from a genetic disorder/disease. There are two gene therapies currently available for me, one of which (the one I'm on) is an oral medication. I believe it uses a virus-like structure to edit a certain gene which is otherwise non-functional in my body.

 

[Rhea]

What’s absolutely amazing is that the results are felt by the patient in just weeks. Weeks. from a debilitating, fast progressing, lethal disease, to “I’m feeling better, I’m thrilled.”

Wow.

 

[Angry Floof]

I knew it! I knew science would find the cure for all the terrible stuff I've been doing to my health for my whole life!

Where do I pick up my new DNA?