The Genetic Engineering Thread

[Anaxagoras]

I think we're about to enter a new era. Sort of like the Internet and smart phones and Google ushered in a new era in the 90s and aughts, we are about to enter a new era of genetic engineering. I don't know exactly what it will all lead to, but I think think the world won't be the same.

Genetically Engineering Pigs to Grow Organs for People

Scientists announce the birth of 37 pigs gene-edited to be better for human transplant.

The idea of transplanting organs from pigs into humans has been around for a long time. And for a long time, xenotransplants—or putting organs from one species into another—has come up against two seemingly insurmountable problems.

The first problem is fairly intuitive: Pig organs provoke a massive and destructive immune response in humans—far more so than an organ from another person. The second problem is less obvious: Pig genomes are rife with DNA sequences of viruses that can infect human cells. In the 1990s, the pharmaceutical giant Novartis planned to throw as much $1 billion at animal-to-human transplant research, only to shutter its research unit after several years of failed experiments.

Quite suddenly, however, solving these two problems has become much easier and much faster thanks to the gene-editing technology CRISPR. With CRISPR, scientists can knock out the pig genes that trigger the human immune response. And they can inactivate the viruses—called porcine endogenous retroviruses, or PERVs—that lurk in the pig genome.

"PERVs", really.

Anyway, it'll be a great world of the future, just like in Black Mirror!!

[Doctor X]

They felt "heterospecies endogenous non-traitorious allele insertions" would be "too gross."

Baka.

--J.D.

[shemp]

But it's not kosher!

[Bruce]

Humans have been genetically engineering ever since we first started farming. Do you think GOD made that corn you're eating?

We're just getting really good at it.

There is a wave of new cancer fighting drugs coming down the pipe that are gene specific designer drugs. These things are really impressive, but they are incredibly difficult to synthesize and analyze. They are composed of a lipid "backbone" to get through the cell wall, an "anchor" to ensure that they are entering the correct type of cell, and a "bomb" that destroys the DNA of the cells from the inside.

It's a wicked and effective strategy. I picture it as giving the address of the bad guy to agent 007, handing him a key to the bad guy's back door, and a discrete C4 for him to plant once he's inside.

From a synthetic and analytical standpoint, its difficult to control. For one thing, the "bomb" portion of the molecule is incredibly toxic until it's attached to the lipid, so you have to make sure every bomb molecule is attached to a lipid molecule. For another thing, any lipid molecule that doesn't have an anchor molecule is useless because the target cell will ignore it. Finally, once the molecule is assembled, it's impossible to analyze because it's way too big to detect by conventional analytical means. Basically, we have to take a portion of the drug and digest it in a way that won't obliterate it's components and then quantify those components. There is no way to directly quantify the assembled molecule itself and determine how much of the pill contains all three active components.

At least not yet. Q is working on it.


Exit strategy? We never said anything about an exit strategy, Mr. Bond.

[Anaxagoras]

Have you heard of the world of "Biohackers"? These crazy people who inject themselves with untested "DIY" gene therapies?

A Biohacker Regrets Publicly Injecting Himself With CRISPR

[Doctor X]

I remain ever amazed at how many people engaged in science cease paying attention.

The answer is that, as some Humble Yet MagNIfIcent Sage noted wisely in his humbly magNIfIcent sagacity, "science" is a "process rather than an attribute." Scientists remain human subject to the same errors all humans can make.

--J.D.

[Anaxagoras]

The CEO Who Injected Himself With An Experimental Herpes Therapy Was Found Dead In A Sensory Deprivation Tank

28 years old. Shame.

Aaron Traywick, a biohacker who once injected himself with an untested herpes therapy on a crusade to expand access to medications, was found dead on Sunday morning in Washington, DC, police confirmed to BuzzFeed News.

Traywick, 28, was found in a spa in Northwest DC, according to police. Staff discovered him in a sensory deprivation flotation tank, according to his colleague Tristan Roberts.

His body was taken for an autopsy, and his cause of death was not immediately known. Their investigation is still ongoing, but the police say they don't suspect foul play.

Traywick was the CEO of Ascendance Biomedical, a tiny, controversial biotech startup on a mission to speed up the process of getting potentially life-saving treatments to patients.

At a biohacker conference in Austin in early February, Traywick made himself a guinea pig, injecting his leg with a never-before-tested, non-FDA-approved, gene-altering herpes treatment made by the company.

“I do what has to be done for the science to move forward and for other people to feel free enough to be able to seek interventions for themselves,” he told BuzzFeed News at the time.

A few months prior, Roberts, another Ascendance employee, had injected himself with an experimental HIV treatment.

Both radical stunts drew crowds on Facebook Live and made headlines around the world. But many watched with heavy skepticism: The company had released scant details about the underlying science of how these treatments might work.

On Tuesday, Roberts described Traywick as a "passionate visionary" and "a warrior for a better future."

"He seemingly never tired as he brought people together to work on some of the most imposing challenges facing humanity," Roberts said in a statement. "While many in the biohacking scene disagreed with his methods, none of them doubted his intentions. He sought nothing short of a revolution in biomedicine; the democratization of science and the opening of the flood gates for global healing."

Shortly after the Austin event, Traywick locked himself in one of the company's labs for several hours and locked out other employees, according to Gizmodo. Some employees said that they had become uncomfortable with Traywick's theatrics and false claims to the press and would no longer be working with him.

And soon after that, Traywick sued Gizmodo and a biohacker, Josiah Zayner, for libel, claiming that they had made false and defamatory statements about the company and its research. Last month, the suit was dismissed, according to court documents.

In the biohacking world, Zayner and Traywick were considered rivals. Zayner — who's done his own self-gene-editing experiments in an attempt to beef up his muscles — had criticized the Ascendance CEO for making unsubstantiated and potentially dangerous claims about treating medical conditions.

"Usually, most biohackers are considered pretty crazy and very controversial, but he was the most controversial of the biohackers," Zayner told BuzzFeed News, adding, "He just wanted to get stuff out there, he didn't care about the consequences to him or sometimes other people. That could be reckless, or it could be good, depending on how it ended up."

[sparks]

"...crazy and controversial..."

Nope. Just dead.

But not as dead as these cigarettes.

[ed]

Humans have been genetically engineering ever since we first started farming. Do you think GOD made that corn you're eating?

We're just getting really good at it.

There is a wave of new cancer fighting drugs coming down the pipe that are gene specific designer drugs. These things are really impressive, but they are incredibly difficult to synthesize and analyze. They are composed of a lipid "backbone" to get through the cell wall, an "anchor" to ensure that they are entering the correct type of cell, and a "bomb" that destroys the DNA of the cells from the inside.

It's a wicked and effective strategy. I picture it as giving the address of the bad guy to agent 007, handing him a key to the bad guy's back door, and a discrete C4 for him to plant once he's inside.

So you could get rid of a population that has some genetic characteristic?

[Bruce]

Some people think that's what AIDS was for...

[sparks]

Nixon had access to Pepto. And he still looked like he was full of shit.

Go figure.

[Anaxagoras]

Time for an update to this thread.

I think we're about to enter a new era.

So I just came across this article:
When a lottery 'wins' sick babies life-saving drugs

Eva was diagnosed with spinal muscular atrophy, a motor neuron disease, just a few weeks after her birth in August. Her parents plan to enter her in a controversial "drug lottery" in the hopes of getting access for a promising new treatment that costs $2.1m (£1.6m) per patient.

The focus of the article is on how expensive it is (over $2 million/patient), and the fact that there's a lottery for people who don't have $2 million to pay for the drug but need it. But I believe the cost will eventually come down. This is a brand new kind of gene therapy. It took a lot of money of course to develop it and to test it, and it's for a pretty rare genetic condition. So I don't look at it as "greedy corporation preys on desperate people" because the amazing thing though is that this kind of therapy exists at all, for any price. And they are in fact, giving it away for below cost to those who cannot afford it.

The Batistas' priority was getting the best care for their newly diagnosed child.

In Canada, where the couple live, they have access to Spinraza, a prescription drug taken during the patient's entire lifetime that can increase survival and motor function. It's been on the market since 2016 and is the first approved therapy to treat all types of SMA.

There is also a brand new drug - Zolgensma. It's produced by Swiss pharmaceutical giant Novartis and was recently approved by the US Food and Drug Administration (FDA) for children under the age of two.

It's a one-time prescription gene therapy touted as a potentially life-changing treatment. It's also currently the world's priciest single-dose treatment, at $2.1m per patient.

Gene therapy, at the cutting edge of health science, involves introducing genetic material into cells to compensate for abnormal genes - in the case of SMA, the faulty SMN1 gene.

In consultations with Eva's doctors, the Batista's have begun treatment with Spinraza - but they see gene therapy as their daughter's best hope.

There is third option for the Batistas - one that depends on the luck of the draw.

When demand from families for Zolgensma spiked following its approval in the US last May, Novartis subsidiary AveXis announced a programme to provide the therapy free to some eligible SMA patients.
Drug becomes 'coveted prize'

It would give out up to 100 doses to patients who meet clinical criteria using a "blinded selection" - a lottery - every two weeks through 2020, starting on 3 February.

The firm said in December that the managed access programme was "anchored in principles of fairness, clinical need and global accessibility...that doesn't favour one child or country over another".

Novartis says the programme was developed with input from bioethicists - but it has been criticised by patient groups around the world.

Kacper Rucinski, co-founder of the UK's TreatSMA and a board Member with SMA Europe, say they will be asking the pharmaceutical firm to remove "fully or partially" the lottery element of the programme.

Is a lottery an unfair way to distribute a limited resource? Anyway, let's hope the day arrives soon when every patient who needs this therapy can have it.

Here's an article about the drug itself:
https://en.wikipedia.org/wiki/Onasemnogene_abeparvovec
And another one about gene therapy, a very new field.
https://en.wikipedia.org/wiki/Gene_therapy

[Pyrrho]

There are other such treatments in development. Scroll down the linked page for the list.

https://www.curesma.org/treatment/

One treatment may work better than another in a given patient, so the more options, the better.

Pricing now...that is a bit more complicated. Suffice to say that any company will charge what the market will bear. SMA is classified as a rare disorder which in and of itself will result in a treatment costing more.

[Witness]

Genetically engineered moth released for first time



Scientists have for the first time released a genetically engineered, self-limiting insect into an open field.

Researchers hope the field test marks the beginning of a turn in the momentum in the war between the diamondback moth, Plutella xylostella, and growers of brassica crops like cabbage, broccoli and cauliflower.

Every year, the diamondback moth, sometimes called the cabbage moth, does billions of dollars in crop damage. Scientists have been searching for a way to combat the pest without resorting to stronger and stronger pesticides.

Oxitec, a British biotechnology company, has developed a solution, a self-limiting moth strain.

In a first-of-its-kind field test, researchers at Cornell University released the company's genetically engineered males to interact and mate with their wild counterparts.

"The moth contains a gene that confers female-specific mortality in the larval stage," lead researcher Anthony Shelton, a professor of entomology at Cornell, told UPI in an email. "When the released males mate with females in the field, they carry the male-selecting, self-limiting gene and the female progeny from that mating do not survive, causing the population to decline."

In previous lab tests, modified males successfully competed for mates, passing along the self-limiting gene and stunting reproduction, but researchers need to be certain the genetically engineered moth behaved similarly in the field.

The results of the field tests -- published this week in the journal Frontiers in Bioengineering and Biotechnology -- confirmed that the Oxitec moth behaves as expected.

https://outline.com/Av4vaP

[sparks]

Life will find a way.

And Jeff Goldblum is watching you poop.