You'll Never Have Full Control

You'll Never Have Full Control

Two of the most fundamental human drives are:


  • The drive to learn more about the world
  • The drive to gain more control over one’s environment and circumstances
  • While these may seem different, they’re connected at the most basic level. By learning more about the world around us, we can figure out how to predict what will happen within it. The more knowledge we accrue, the better our predictions become, and the better we’re able to prepare for what were previously unforeseen curveballs. A life of proper prediction and diligent preparation allows us to feel stable and secure. Of course, our predictions are less than perfect, and we’re often wrong with disastrous consequences (as the works of Nassim Taleb and Daniel Kahneman illustrate). But most of our life actions, and much of the technology that we produce, have the sole aim of increasing our feeling of control.

    Uber is a great example of this. Traditionally, finding a taxi has been a bit of a crapshoot. While you may be lucky from time to time, and flag down a taxi within a minute of stepping up to the curb, it’s not uncommon (especially in San Francisco) to have to spend 15 hand-flapping minutes at the corner of a busy thoroughfare before successfully ensnaring a cab. With Uber, however, you are practically guaranteed a ride each and every time you open the app. You can see who your driver is, what model of car they’re driving, and where they are on a map in relation to you. Location is accurate up to the second. This is a prime example of a human-made system with the sole purpose of giving common people more control over their circumstances. Stated differently, it’s a great example of a human-made system with the purpose of reducing uncertainty.

    As I’ve written about before, if you look at the technologies we’ve produced since the beginning of history, all of them have taken what was once a scary, indeterminate part of life and made it mundane and determinate. Farming took the hunt out of hunting and gathering. Medicine, vaccines in particular, took the fear out of common sicknesses and ailments. Communication technologies gave people instant feedback of whether their communication was received (and successful). Each new successful innovation will do the same thing, and wring a little bit more uncertainty out of a part of life.

    Medical innovations will continue to make the probability of death, at any given age, closer and closer to zero. Transportation technologies will continue to make the probability of getting transported safely and quickly higher and higher. And dating applications will continue to get better and better at making the possibility of meeting up with a “dud” closer and closer to zilch. Our locus of control will continue to increase throughout the 21st century, extending out our human powers beyond our wildest dreams. Unfortunately, as our locus expands beyond our skin, and begins to encapsulate the world, we’ll come to find ourselves simultaneously in control of everything and controlled by everyone else. We’ll be back where we started. Even if we squeeze the uncertainty out of life, there will always be messy, chaotic people to foil our plans. After all, there is no app for making old, painful memories fade away, or for quelling the heartache of a love lost. At that point, all we can do is learn and move on, and let wisdom reduce uncertainty within when there’s nothing more to do without.

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    For the first time, researchers appear to have effectively treated a genetic disorder by directly injecting a CRISPR therapy into patients' bloodstreams — overcoming one of the biggest hurdles to curing diseases with the gene editing technology.

    The therapy appears to be astonishingly effective, editing nearly every cell in the liver to stop a disease-causing mutation.

    The challenge: CRISPR gives us the ability to correct genetic mutations, and given that such mutations are responsible for more than 6,000 human diseases, the tech has the potential to dramatically improve human health.

    One way to use CRISPR to treat diseases is to remove affected cells from a patient, edit out the mutation in the lab, and place the cells back in the body to replicate — that's how one team functionally cured people with the blood disorder sickle cell anemia, editing and then infusing bone marrow cells.

    Bone marrow is a special case, though, and many mutations cause disease in organs that are harder to fix.

    Another option is to insert the CRISPR system itself into the body so that it can make edits directly in the affected organs (that's only been attempted once, in an ongoing study in which people had a CRISPR therapy injected into their eyes to treat a rare vision disorder).

    Injecting a CRISPR therapy right into the bloodstream has been a problem, though, because the therapy has to find the right cells to edit. An inherited mutation will be in the DNA of every cell of your body, but if it only causes disease in the liver, you don't want your therapy being used up in the pancreas or kidneys.

    A new CRISPR therapy: Now, researchers from Intellia Therapeutics and Regeneron Pharmaceuticals have demonstrated for the first time that a CRISPR therapy delivered into the bloodstream can travel to desired tissues to make edits.

    We can overcome one of the biggest challenges with applying CRISPR clinically.

    —JENNIFER DOUDNA

    "This is a major milestone for patients," Jennifer Doudna, co-developer of CRISPR, who wasn't involved in the trial, told NPR.

    "While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place," she continued.

    What they did: During a phase 1 clinical trial, Intellia researchers injected a CRISPR therapy dubbed NTLA-2001 into the bloodstreams of six people with a rare, potentially fatal genetic disorder called transthyretin amyloidosis.

    The livers of people with transthyretin amyloidosis produce a destructive protein, and the CRISPR therapy was designed to target the gene that makes the protein and halt its production. After just one injection of NTLA-2001, the three patients given a higher dose saw their levels of the protein drop by 80% to 96%.

    A better option: The CRISPR therapy produced only mild adverse effects and did lower the protein levels, but we don't know yet if the effect will be permanent. It'll also be a few months before we know if the therapy can alleviate the symptoms of transthyretin amyloidosis.

    This is a wonderful day for the future of gene-editing as a medicine.

    —FYODOR URNOV

    If everything goes as hoped, though, NTLA-2001 could one day offer a better treatment option for transthyretin amyloidosis than a currently approved medication, patisiran, which only reduces toxic protein levels by 81% and must be injected regularly.

    Looking ahead: Even more exciting than NTLA-2001's potential impact on transthyretin amyloidosis, though, is the knowledge that we may be able to use CRISPR injections to treat other genetic disorders that are difficult to target directly, such as heart or brain diseases.

    "This is a wonderful day for the future of gene-editing as a medicine," Fyodor Urnov, a UC Berkeley professor of genetics, who wasn't involved in the trial, told NPR. "We as a species are watching this remarkable new show called: our gene-edited future."

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