Two US Patients Treated With CRISPR Gene Editing

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The University of Pennsylvania confirmed for the first time that two US patients had been treated using CRISPR gene editing.

Several other human trials of CRISPR are starting or are set to start in the US, Canada and Europe to test CRISPR’s efficacy in treating various diseases.

The work removes immune system cells from patients, genetically modifying them in the lab and infusing the modified cells back into the body. Gene edited t-cells to fight cancer has been performed extensively using zinc finger gene therapy.

Sangamo Therapeutics has been developing Zinc finger nuclease (ZFN) autologous and allogeneic gene-edited cell therapies for blood disorders including beta-thalassemia and sickle cell disease, immunological disorders, as well as various types of cancer. Sangamo has been studying zinc finger nucleases for over 20 years and is the primary patent holder for this technology. CRISPR gene-editing emerged in 2013.

The primary criticism of zinc finger nucleases as a gene-editing tool is the time and skill it takes to develop a high-quality gene snipping product compared with CRISPR. Sangamo has shortened the development timeline for zinc finger nucleases.

CRISPR is less flexibility for target any gene sequence. Target gene sequence for CRISPR must be immediately upstream of a protospacer adjacent motif (PAM). Zinc fingers have no restriction of that type.

CRISPR is much faster and cheaper to work with in the lab.

Two CRISPR trials sponsored by CRISPR Therapeutics and Vertex Pharmaceuticals are designed to treat genetic blood disorders. One is for sickle cell disease, and another is a similar genetic condition called beta thalassemia.

Editas Medicine will try to treat an inherited form of blindness known as Leber congenital amaurosis. Editas will try to edit genes inside the human body.

19 thoughts on “Two US Patients Treated With CRISPR Gene Editing”

  1. You said it all when you said “we have in the immediate future”.
    The future is coming much faster than you think! Anything is pretty much literally possible beyond your wildest dreams and soon.

  2. “I think ,I have not been expressing my thoughts clearly.”
    Oh, you have. I just disagree.
    That’s because anyone that has been practicing medicine- specifically any laboratory medicine that deals with genetic aberrations with gross manifestations- knows that you can talk about flipping genetic switches as much as you want. But at the end of the day, sometimes you have to just manually change things yourself[grossly].
    Because, as I have been imparting- some of this requires far too much genetic manipulation for what we have in the immediate future to just flip switches. Because long term effects are sometimes just permanent.

    That’s the part I am telling you that you are not listening to.
    Again, look at examples of people with OI. Those effects are permanent and I cannot think of anything on the near or distant horizon[not in the pipeline or has even been fathomed], from a genetics standpoint, that will change it.

    If you have specific example[and I mean REALLY specific, down to the specific cell lines that are going to be manipulated by specific genes and how you are going to get around pre-laid down woven bone on a gross level; beyond simple osteoclast/blast turn over] then by all means.
    Or do it with gigantism. Or Turner Syndrome. Or whatever. Cases like Cystic Fibrosis are simple. But some of these conditions are lifelong, regardless of genetic alterations you make. You’d have to manually change someone’s phenotype WITH it.

  3. Greater intelligence would be nice, but I was pointing out what we can do with our current intelligence in the meantime.

  4. I think ,I have not been expressing my thoughts clearly.
    We are biological/chemical machines. With a biological nano factory built in.
    That nanofactor can build just about anything if it has the right blueprint(DNA). It can disassemble, make changes and then reassemble any part of the body, a nano part at a time. That includes bones, muscles, nervous system, brain etc.
    It has the ability to build parts with molecules that are not even part of living matter.

  5. I am listening and intensely. It’s just that I believe any changes the body makes can be reversed. Maybe not with the genes we have now as humans but altering those genes in precisely the right way can achieve almost anything.
    For example telepathy or at least a similar effect. A electric ell can produce huge voltages and broadcast them at will. So by harnessing their genetic ability coupled with a electric receiver similar to what sharks have but much more sensitive we could communicate with telepathy. I know this sounds far fetched but that is what the science fiction writers were trying to convey to us with their bio/living ships. Using biomass controlled by genes we will be able to produce bio circuits with similar results to today’s electrical circuits.

  6. I used quotes. But you’re not listening. You’re not going to get taller that way.
    Again- the growth plates have already ossified. You would have to grossly change that.
    In very young kids? Yea, sure. You can regulate growth plate ossification with hormonal regulation, alone.
    Your height mostly comes from long bone lengthening. After a certain point, usually after puberty, the growth plates ossify and that is typically your final height.

  7. I never used the word bigger, I said faster or taller. I am not a human biology expert but I do know that bones can both heal and grow (alter shape) into adulthood. Besides with genetic engineering the bone can be reshaped. If other animals can do it with their DNA, it can be done with ours.(and much more)
    Yes, it is incredibly complex! Personally I think that only quantum computers will be able to figure it all out.

  8. Are you speaking as a cro magnon man against the enhancement of all of our intelligence?
    As we are now we are not smart enough to really see the possible negative consequences of our attempts to alter our atmosphere. We could inadvertently make matters much worse.

  9. No, it’s not that simple. The bone plates have already ossified. So it doesn’t matter that you do after that.
    You turn on the genes to “Get bigger”? Yea, that’s called gigantism and can be caused by pituitary adenomas. You’re going to get an enlarged head, hands, feet and organs and probably die of heart failure. Your vertical growth will be there but not like you think.
    DNA being the “blueprint of life” is only part of the story.
    Same with OI- you’ve already laid the mature bone down and it is brittle. You would need to do gross scaffolding WITH genetic engineering to fix something like that, even ignoring that the neural plasticity for moving body parts may not be exactly what it should in a normal adult.
    You go in there and just fix a defective collagen gene, then you start having fibroblasts pumping out collagen in an ECM they normally shouldn’t and you could start having problems.

    My point is that this is not as simple as turning on a genetic switch. The body matures in a certain way and it is incredibly complex and is more than just genes.

  10. Replace fossil fuels with nuclear for electricity would cut CO2 emissions drastically. Try Ocean Iron Fertilization to see if that will safely remove a substantial part of what has already been emitted.
    Why do you think immensely greater IQ is needed, when the brighter ones already among us can see reasonable solutions?

  11. We are a soon to be a extinct species. We cannot reverse Climate change fast enough to prevent extinction as we are now, even if every man woman and child pulled together. The reason for the need for speed is that with a IQ of 10 fold, you personally would know how to re terraform the EARTH quick enough to prevent our extinction. Plus other benefits like fusion. What if we were all genetically altered to be 1/4 as big as we are now, The resources we would use would be proportionally smaller. Many will argue that we need more wisdom not intelligence. I argue that wisdom is a byproduct of intelligence. We cannot compare any two men’s wisdom to see this, there is just not enough difference in intelligence between men. (not even Einstein). In general a ant is wiser than a bacterium, a dog is wiser than a ant. A chimpanzee is wiser than a dog and a man is wiser than a chimp. Hopefully with a 10 fold increase in intelligence we will be much wiser to.

  12. DNA is the blueprint of life. Any change is possible. For example to grow taller the genes would just be given the instructions to grow taller or the bones straighter. This as you know would take a long time, But wait, how fast a life form grows is genetic based to. So we just speed the process up. The only limitations if there are any, are the laws of physics and speed of chemical reactions. I know how far fetched this all sounds but once YOUR intelligence is stretched 10 fold, you will personally be able to do much more amazing things than these.

  15. I think that’s selling it a little too much, or do you have a reason we absolutely need it very quickly, compared to it being a very good thing but not critical?

  16. Early onset genetically based diseases, sure.
    But some people afflicted with certain diseases may have permanent effects. By way of example- osteogenesis imperfecta.

  17. Perhaps not this time but of course there will be negative consequences. Some will be very terrible but the whole future of the human race depends on this technology being developed quickly.

  18. This is the start of cures for ALL diseases. And with generation 2 in the near future your genes will be engineered to prevent all disease.
    Generation 3 is where it gets interesting. A genetic intelligence boost to ALL humans.
    Refusing treatment will be like a very early cro magnon man not wanting to be a modern human. (If he could even comprehend the concept).
    As far as cost in the future, you can buy gene engineering kits on line to experiment with. The new gene sequence will be like a modern day print for a 3d model. The model itself is not worth much because anyone can print one.

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