The Vast, Bloody Gap Between Theory And Practice

The Vast, Bloody Gap Between Theory And Practice


Everyone has a large number of great theories or ideas. Here’s one that I have: Wouldn’t it be great if all of the money that each person generated was split up and distributed evenly across all of the members of their family and community? That would increase a feeling of connection and camaraderie, and reduce the burden that each member of the community would need to bear. Sounds good, doesn’t it?

The only problem is that it ignores all practical experience and knowledge about how people actually behave. People are motivated by status and material gain and, if they don’t receive the full fruits of their labors, they’re not going to work as hard or produce as high quality work. Thus, if all money were distributed, there would be less wealth creation and a general feeling of malaise and discontent in the community. Why the malaise? Because we gain a great deal of our self-regard and meaning from a job well done. There are very few couch potatoes that are as happy and content as an Elon Musk or an elementary school teacher. This is simple common sense, something that everyone outside of a small sliver of pie-in-the-sky academia knows. It’s what I call grandma wisdom: something so long and universally known that even your grandma could tell you about it.

However, imagined scenarios are rarely, if ever, at full fidelity, and so a lot of important information is left out of these utopian fantasies. For example, these visions never spell out how likely it is that the parties in question will conform to the scheme in question, and they never opine on how the changed incentive structure will cause the behavior of the actors to change – which often nullifies the desired result.

A wonderful illustration of this error in thinking can be seen with former New York Mayor Bloomberg’s proposed soda ban. He wanted to decrease the amount of sugar that individuals in New York could drink, so he pushed to ban sodas above a certain size. The thinking was as follows: If people are not able to purchase 16oz+ drinks, they will purchase 12oz drinks, thus consuming 50 fewer calories at mealtime. The “problem”, however, is that people are not simple automatons that mindlessly execute the same behavioral script over and over – they change according to new wants, needs, and conditions. 16 ounce sodas are no longer available? Okay. I’ll purchase two 12 ounce sodas instead. Thus, instead of diminishing the grams of sugar that individuals would purchase and consume, this law would have the perverse effect of increasing the sugar calories consumed.

Impractical thinking like this wouldn’t be a problem if these fantasies were locked in the heads of their creators. The problem is that the creators take them seriously, and often try to translate these ruminations into reality, which almost always results in disaster. The 20th century is a monument to the danger of theoretical utopian thinking, and the world is 100,000,000 people poorer today because of it. The attempts of optimistic leaders to jam Communism down the throats of their peoples should be a lesson and a warning for us all: human nature doesn’t conform to the shape of the container into which it’s forced. Instead, it either perishes or revolts with disastrous consequences.

Image: Anthony Easton

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This article was originally published by our sister site, Freethink.

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.

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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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