CRISPR used for the first time to edit DNA still in a person's body

I find all the CRISPR news really fascinating. I guess scientists were recently able to edit someone’s DNA without removing it.

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.

Francis Collins, director of the National Institutes of Health, calls the advance “a significant moment.”

“All of us dream that a time might be coming where we could apply this approach for thousands of diseases,” Collins tells NPR. “This is the first time that’s being tried in a human being. And it gives us hope that we could extend that to lots of other diseases — if it works and if it’s safe.”

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It is worth noting that Dr. Francis Collins was extremely vocal about his ethical concerns when a Chinese scientist edited the genome of human embryos. His support of this specific study is well earned, and it helps to define how this technology should be used in the future. I also agree with Dr. Collins that there is a lot of hope around this technology if it works and if it is safe. I also think the doctors in this most recent case are on a solid ethical foundation because the benefits outweighs the risks, there is informed and open consent, and the technologies used were carefully and wisely chosen.

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Right, there is clearly a major ethical difference between editing somatic cells and editing germlines.

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This is really cool. I just hope the technology can get verified and quickly manufactured and made affordable. Sometimes after seeing a single pill’s price jacked up it just makes you wonder how high could they drive a price. Definitely should be balanced between profitable and affordable.

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I have a lot of lab experience working working with DNA recombination techniques, but I don’t have much experience in the pharma or medical fields. With that said, here are my thoughts.

Leaders in both government and medicine should get ahead of this issue and adjust patent law so that specific gene edits can not be patented. It would also be helpful to reduce the costs for CRISPR clinical trials through some sort of standardization. If these steps were taken then there could be healthy competition between many different providers which would drive prices down.

The development of these treatments is very different from traditional drug development, at least from what I can see. The first and hardest step is identifying the genetic cause of the disease. The next and less difficult step is identifying a gene editing strategy that could restore healthy function. The last and relatively easiest step is designing the CRISPR genes for the edit. Traditionally, designing the drug is the hardest part, but that doesn’t seem to be the case with CRISPR.

Essentially, companies would be offering a service, not a drug. If regulated properly, we could see a relatively (add in heavy air quotes) affordable market for CRISPR treatments. Companies would make money on every patient through the services they offer without needing to patent a single drug or worry about a short patent window to profit off of their drug before it goes generic. I am probably being way too optimistic, but I do see a way that this technology really could be both affordable and transformative.

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At least we seem to be leaps and bounds more ethical then we were just a century ago where people were locked away for suffering from feeblemindedness and routinely people were used as test rats for surgeries, electro therapy and we forced undesirable or handicapped people into the tortures of the “forward thinkers” who committed eugenic atrocities. It’s craziness.

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