Many CRISPR Gene Editing Clinical Trials Underway

Human clinical trials are using CRISPR/Cas9 to combat cancer and blood disorders. In these tests, researchers remove some of a person’s cells, edit the DNA and then inject the cells back in, now hopefully armed to fight disease. Researchers are also set to see how CRISPR/Cas9 works inside the human body. In an upcoming trial, people with an inherited blindness will have the molecular scissors injected into their eyes.

Future trials for Duchenne muscular dystrophy, cystic fibrosis and a wide variety of other genetic diseases will help millions of people worldwide.

University of Pennsylvania researchers have given two people with recurring cancers a CRISPR/Cas9 therapy. One person has multiple myeloma; the other, sarcoma. As part of an ongoing trial, both received T cells, a type of immune cell, programmed with CRISPR to stop cancer cells.

A clinical trial using CRISPR is treating an inherited type of blindness called Leber congenital amaurosis 10. A mutation in the CEP290 gene that leads to a nonfunctional protein. When the protein doesn’t work, rod cells in the retina die and light-gathering photoreceptors can’t renew themselves, resulting in blindness.

Editing as few as 10 percent of retinal cells may help restore some sight. In animal tests, CRISPR edited up to about 60 percent of cells in mice and almost 28 percent in monkeys, scientists reported in the February Nature Medicine.

In July, Editas and global pharmaceutical company Allergan opened recruitment for a blindness gene-editing trial. In the trial, two guide RNAs will lead Cas9 to make two cuts that will snip out the troublesome piece of DNA.

40 thoughts on “Many CRISPR Gene Editing Clinical Trials Underway”

  1. There are definitely different levels of immortality.

    1) Limited immortality: Not being subject to aging
    2) Lesser Immortality: Being able to survive the instant and unexpected destruction of your current body and/or brain
    3) Greater Immortality: Being able to survive the instant and unexpected destruction of your planet (space habitat, whatever).
    4) We can conjecture the next step might be to be able to survive the instant and unexpected destruction of your star system (although speed of light issues might require some really iffy definitions on what survive really means).

    I actually encounter this problem in some of my projects. The client wants “real-time” updates but then we have to wrangle on what that really means. A lot of times they are quite happy to settle on intervals of fifteen minutes, hourly, or even daily, rather than something measured in nanoseconds.

  2. No tablets, that was too old fashioned for him, that was for the old Syrians. No, he wrote his messages on vellum.

  3. I am somewhat overtired, when I saw the picture, My initial thought was: oh, the pope is cutting his rosary. 😉

  4. Copy or original, knowing that there are several perfect copies of me won’t increase my propension to risk even a little bit.

  5. > the copy will remember making the tatoo, but won’t have it on its body.

    Unless the tatoo is also duplicated. Not having it duplicated is the same as “being told”.

  6. > […] omozygotic twins who share the first part of their lives

    That’s one way to look at it, but a little biased by our current experience of only having one body.

    If the copies periodically (or continuously) sync, it’s harder to draw the line between them. Then they can be considered parts of a larger whole. Like a mini-hive-mind inhabiting several bodies.

  7. Unless doing a backup requires you to sleep,
    of course. In that case, make a tatoo on your body before the backup, the copy will remember
    making the tatoo, but won’t have it on its body.

  8. The copies are equivalent only at the moment
    of the backup, then they start to diverge.
    Different experiences, different persons,
    omozygotic twins who share the first part
    of their lives.

  9. In a scenario where you have multiple identical copies running around and periodically syncing back, even if you don’t consider them “you” and keep the “main you” in a bunker, you can still experience the risky stuff via the copies.

    They don’t have to sync in real-time, so can be autonomous enough to have unlimited range. Actually, if they’re perfect copies, they will be autonomous. But then, they’ll think they are you, and have the same personality, so they’ll probably join you in that bunker. Some accounting and coordination would be needed to prevent that.

    My personal view is if the copy is accurate enough, than it is you. That’s because unless you’re told, none of you (nor anyone else) can tell which one is the original. You’d all have the same memories, same personalities, identical bodies, and you’ll all be equally convinced each of you is the original. So all of you would be equivalent.

  10. No, a perfect copy of myself is myself only for itself and the others. I would put my brain in a
    NORAD-style bunker, wirelessly linked to physical
    (or virtual) bodies, which would not need to be longeve and could be changed and risked at will,
    at least if I have enough money. Only problem,
    they probably couldn’t move more than a few
    hundred miles from the brain. Where there
    should be the brain, those bodies would house
    the comm equipment and the interface with the peripheral nervous system.

  11. > half-life of a human population would be just about one thousand years

    If you can upload, you can take backups. So if you die, you can restore from a recent backup. If you accept that multiple bodies (or an upload) are all “you”, then backup-restore isn’t much different.

    Furthermore, with multiple bodies you can have some of them take risks while others stay safe. You can periodically split off new copies to replace old ones or ones lost to accidents, and “spread your eggs” so that the chance of all your copies getting destroyed simultaneously is much much lower.

    I’d imagine that would extend the half-life quite a bit.

    edit: Come to think of it, it’s similar to how individual cells and life-forms only live for very short times (up to a few thousand years for long-lived trees), but the cell-line as a whole can survive for billions of years by periodically splitting off copies (offspring). The difference is here your copies/”offspring” share your personality and memories – and as a result, your identity.

  12. Currently, a little math shows that if people could live without aging or disease, the half-life of a human population would be just about one thousand years.

    Those that were older than that would probably be the ones that were far less likely than the starting population as a whole was to do things like take up extreme sports, travel, work with animals, or even leave their homes.

    Eventually the only ones left would be the really superlucky ones (and that run of luck can always end as quickly as coin flip) or the ones that live in asteroid proof bunkers with heavy security and never come out except maybe in VR (assuming NEO and Agent Smith can’t kill you there) or in suro bodies.

    And, of course, their accrued wealth and power would probably assure they are the ones running things.

  13. Lobots would be cheap chinese robots, that fits. I would insist on
    paleo appearing body forms regarding girls, who would obviously
    be cute, compliant andies. The key questions are those regarding
    the brain, the body is only a vehicle. Of course, if cell senescence is
    stopped, risk avoiding becomes paramount, and the brain should
    be protected at all costs. That might be the reason of the Fermi
    paradox. Based on our experience, life either has no wireless, or is
    superAI directed. On geological, or even historical timescales, intertime is a blink, less than two centuries.
    “SuperAI, what should we do regarding alien intelligence”
    SuperAI: ” Alien SuperAI might be horribly dangerous, so let’s
    keep our heads down.”

  14. In the U.S.

    Heart disease 23.4%
    Cancer 22.5%
    Smoking 5.6%
    Accidents 5.2%
    Strokes 5.1%
    Alzheimer’s 3.6%
    Diabetes 2.9%
    Flu 2.1%
    Kidney disease 1.8%
    Suicide 1.6%

    I kind of expect that, fifty years from now (in developed countries with not too much socialism at one extreme, or income inequality at the other), and if a new cause does not arise, suicide or accidents will head this list, but not because they will have increased in frequency per 100,000, but because the other eight will be much reduced.

    Saw an article recently that said humans are the only animal subject to heart attacks and that it has to do with some mutation that improved our chances against malaria but causes no end of trouble in our bloody plumbing.

  15. Heh, would expect Avis or Hertz to rent out the units to Angels looking to make a temporary switch in status. Otherwise, they might get a multi-year lease from the manufacturer if they don’t want to buy one outright.

    Unless there is a perfect storm of life extending technologies available for my (currently) mod body in the very near term (and there might be but I’m not holding my breath), I don’t expect to see much, if any, of this in my lifetime.

    Categorizing comes down to the answers to some basic questions. Does the individual:

    1) Allow regeneration, rejuvenation, organic replacement, or inorganic replacement? Which ones, if not all of them?
    4) Insist on paleo appearing body form? Or something similar?
    2) Allow upgrades or just replacements? Can they be visible?
    3) Allow cognitive upgrades and replacements in addition to physical?
    5) Have a mind that originated as paleo, lifted animal, or synthetic?
    6) Have a mind that’s been altered? How? Also, where is it kept?
    7) Primarily exist in the “real” world, VR, or a mix?
    8) Have any self-imposed limits on upgrades, especially cognitive?
    9) Have a mind that is multi-threaded, exists in multiple bodies, or have multiple instantiations (that would periodically merge back together)?

    This is off the cuff, I’m sure I’m missing many.

  16. Ancient beliefs about magic included things we can trivially achieve with modern tech… and also included stuff like eternal youth, restoring the dead to life, lead into gold etc. etc.

    It isn’t that they were less than what we have achieved. Or more than what we achieve. It was really quite different from what we have achieved.

  17. A Suro should be able to become an Angel at the flip of a switch. Let’s hope in the
    Omega Point, but pessimism prevents delusion.

  18. To tell the truth, advanced technology is already better than
    medieval magic, which is something stuck in between herbal
    remedies and the steam engine. King Arthur would have never
    dreamed of a tablet.

  19. Inorganics:
    Hi-bots – robots, can look like anything (other than Paleos), can pass a true Turing test, have citizenship
    Andies – hi-bots that look completely Paleo
    Bottybots – hi-bots, but inorganic mind is elsewhere (operated remotely)
    Surobots – andies, but inorganic mind is elsewhere (operated remotely)
    Transcenders – regardless of origin or composition, pushing limits attainable in a single physical body
    Legos – regardless of origin, multiple copies of minds (and often bodies) periodically remerged.
    Virts – regardless of origin (Angel or Artilect), lives entirely in virtual reality, and is now fully inorganic
    Godlings – regardless of origin, unconfined by any limits on body form, and push the absolute mental limits, if any exist
    Hiveminds – networked minds of any origin, embodied or not
    Lifts – animals upgraded to pass a true Turing test
    (Can also be a potential subcategory of certain categories, e.g. borglift, surolift, and ceases to matter at trans levels)

  20. It helps introduce a complex future. Some time back, I tried to categorize some of the possibilities.

    Paleo – Essentially 20th century unaltered humans
    Christies – Paleos that may need modern medical procedures, but will not permit it
    Amies – Paleos that permit any or most medical procedure (but not upgrades)
    Pervies – Amies that either will allow organic but not inorganic replacements (or the reverse)
    Clarks – Paleos that actively seek, and have received, numerous organic upgrades
    Mods – Paleos with minor implants, to include upgrades, not visible
    Borgs – Paleos with visible inorganic implants, especially if they are upgrades
    Innies – Fully robotic bodies, no attempt at Paleo appearance (but includes an organic brain)
    Synths – Fully robotic bodies, look completely Paleo, (but includes an organic brain)
    Suros – A synth or innie, but an organic brain is elsewhere (operated remotely)
    Angels – lives entirely in virtual reality, but an organic brain is elsewhere (operated remotely)
    Lobots – robots, can look like anything, insufficient state of awareness for citizenship, property
    Bots – lobots that meet or exceed mental capabilities of Paleos, but only in a limited sense
    Apps – applications, a bodiless bot, insufficient state of awareness for citizenship, property
    Artilects – applications that exceed mental capabilities of Paleos, but only in a limited sense
    (Continues in reply)

  21. > genetic engineering is the be all, end all of human advancement

    Only if you consider biology to be the be-all, end-of of human existence. And even then, seems a little short-sighted.

    > Man’s greatest achievement will be using genetic engineering to increase EVERYONE’S intelligence 5 fold or even greater.

    The same could possibly be done with BCIs. The race is on.
    (Note: the two aren’t mutually exclusive.)

  22. I personally believe the obstacles you mention are too great to overcome without a AI(Not self aware!) research system based on a soon to be fully developed universal super quantum computer. Which will happen in the near future.

  23. Don’t make fun of D Drake, his eyes will be open in the near future. Some people just can’t grasp what the facts of exponential growth of technology entails.
    After all it has been said “Advanced technology will seem like magic”.

  24. In vivo gene editing (inside the body, not on cells removed and then put back in) may be possible thanks to the discovery that transposons (jumping genes) have themselves hijacked CRISPR-cas systems in bacteria to insert themselves in multiple parts of the bacterial genome.

    If it pans out, this will be a particularly important development for SENS Research Foundation style anti aging research as certain therapies such as moving the 13 mitochondrial genes to the nucleus will have to be done on long lived cells inside the body e.g. muscle fibers/neurons.

    “However, a long checklist must be completed before clinical applications can be considered seriously. This list includes: showing that the process works efficiently at target genome positions in disease-relevant human cells (rather than in bacteria); demonstrating that it can integrate DNA fragments large enough to be clinically useful; proving its specificity in the human genome, which is about 1,000 times larger than a bacterial one; and developing ways to deliver the full complement of proteins associated with the integration process to cells without triggering the human immune response. This is a formidable workload, but a key lesson of the past 30 years of research into gene therapy is that most challenges of this type are eventually solved. Therefore, a CRISPR system used by transposons to propagate themselves might well find itself repurposed for genetic medicine.”

  25. People who believe crazy nonsense will continue believing crazy nonsense after drinking your milk.

    You still possess a cartoon understanding of intelligence.

  26. BTW this technology is coming unbelievably fast! You can buy gene editing experimental crispr kits on ebay for under $300.
    They are doing a lot of experimental gene editing on domesticated dogs. The people bring their pets in, where they receive treatment and then are sent home again. I understand it only takes a year to get FDA approval for a drug to treat animals. Example – If they make a major breakthrough on a treatment to cure ALL cancers in dogs( that worked equally well on humans) how long would it take before the people demanded the FDA approve it for human use almost immediately?

  27. This is the spectacular future of mankind. Guided evolution. Mankind version 2.0
    While stem cell therapy is really great and a up and coming thing that will lead to many cures and tissue regrowth, genetic engineering is the be all, end all of human advancement.
    It makes no difference whether your for it or against it, genetic engineering is progress and no one has stopped progress in the history of the human race. Occasionally temporarily slowed progress but never stopped progress.
    The greatest achievement of mankind will not be space travel, immortality, fusion etc. Man’s greatest achievement will be using genetic engineering to increase EVERYONE’S intelligence 5 fold or even greater.
    There are plenty of conspiracy theorists that will find the dark side of this and others that say we will become monsters etc.
    If I snuck a liquid in your milk that would raise your intelligence by 5% would you know it tomorrow? NO, you would just be a little sharper. If I did that the next week again so now your 20% more intelligent would you be a monster?
    To fight the inevitable would make you like a cro magnon man saying I don’t want to live to be a modern human and see my children live to 80 years and not die of starvation, cold, or eaten by predators.

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