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.
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."