CRISPR Used To Edit Genes Inside a Patient With a Rare Form of Blindness (npr.org) 25
An anonymous reader quotes a report from NPR: For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body. The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored. Before this step, doctors had only used CRISPR to try to treat a small number of patients who have cancer, or the rare blood disorders sickle cell anemia or beta-thalassemia.
In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery. Beginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement. The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells -- enabling patients to regain at least some vision.
In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery. Beginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement. The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells -- enabling patients to regain at least some vision.
Good Luck - Downsides? (Score:2)
Good luck on the safety and effectiveness. I mean that non-ironically. I hope for the best for them
Now what about their children? The ban on germ-line editing in many nations means that these kinds of diseases will never be 100% eliminated until that is done. That is my long-term concern. Without that, there will always be a new way to make money on this treatment, rather than eliminating the cause for good.
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I don't think the ban on germ-line editing will hold indefinitely. Once the tech, and our understanding of it, is more mature, it will start making sense to allow exceptions to the ban. And then, finally, the streets will run rampant with mutant human-animal hybrids.
Yay for science!
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I for, for one, welcome our new human-ant overlords!
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Life span of the Great Basin Bristlecone pine tree, please.
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So much potential (Score:1)
We're living in the future (Score:2)
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NO one seriously thought we would have flying cars(as displayed). They were a metaphor for the hope of the future.
And talking about and trying to figure out how to rewrite DNA has been talked about and worked on for 25 years.
One of these is literally impossible.
The other is chemistry.
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Technically speaking, flying cars are not impossible. In fact, if you are enough tolerant about what a car is... a modified helicopter could be considered a "flying car".
Of course, they are simply too expensive and even if we omit the cost argument, they are very impractical to use.
At the end, that's the real reason. Any flying thing has a non negible risk to go down, if it does the damage is big and the cost are obviously greater than something that displace on the surface. 3D structure is possible trhough
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I see (Score:3)
"They hope to know within weeks whether the approach is working"
When he walks the corridors and eats the brains of people?
Re:I see (Score:5, Funny)
When he walks the corridors and eats the brains of people?
While this is an unfortunate side effect, I'd like to point out that the capture rate he managed proves how much better his vision is now, compared to the average human's.
No! (Score:2)
"For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body. "
Actually no, They did it the very same way all the other doctors did it before them. Outside the body, then injected it.
from TFA: ....microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery..."
"injected
Sorry, but when I read TFS I just _had_ to RTFA, I apologize.
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You didn't understand what you were reading. They edit a virus outside the body to carry the CRISPR editor into the cells. Where the CRISPR editor edits the genes INSIDE the body.
Other gene therapies have been used to edit the patient's cells outside the body, then those edited cells are injected back into the body. But that's not what they're describing in this article.
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But he needed to show everyone how smart he is with his contrary nature.
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And yet you still missed it.
"ene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body."
and
"deliver the instructions to manufacture the CRISPR gene-editing machinery"
The virus builds the machine, and then the new CRISPR machine(build be the virus in the body), edits the gene inside the body.
Your rush to appear smarter then everyone caused you to look foolish.
The PAtient (Score:2)
The patient was quoted as saying: "MUAHAHAHHAHhahahha" before blasting a nearby building with their laser eyes and then flying off.
Scientist say procedure was a success, and they look forward to serving our new overlord.
That's half the battle (Score:2)
I'm not familiar with this illness, but fixing the retina is part of the battle. The other part is getting the brain to begin processing the received neural impulses as visual imagery again. The earlier and longer the patient has been blind, the more difficult that might be. The article mentions that younger children might benefit more because their 'visual pathways are still intact', but is that referring to the optic nerves or--my supposition--that their visual cortex is better able to restore normal visu
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just in time debugger (Score:1)