CRISPR-Cas9 is the cutting-edge genomic technology that essentially lets you target exact sequences in DNA and then cut into them like a knife and insert or remove a gene. You may remember it from that Chinese scientist who successfully (but controversially) implanted gene-edited embryos into a woman who then gave birth to live lab babies. More often, it's used to create things like malaria-resistant mosquitoes or mushrooms that don't brown as fast.
But it does have a lot of practical medical potential, too. It's already been used to remove HIV from a patient's genome. And now, after CRISPRing out a blindness-causing gene from mice, scientists have now successfully scaled-up this procedure to work in a live human body. From NPR:
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In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery.
The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells — enabling patients to regain at least some vision.
The procedure, which takes about an hour to perform, involves making tiny incisions that enable access to the back of the eye.
In the first injection in a human being of macromolecules whose primary structure was developed from a religious text a French 16 year old named Adrien Locatelli describes how he paid Vector Builder $1300 to transcode verses from the Bible and the Koran into macromolecules and then injected one verse into each leg (the Bible verse was written into the DNA of an adeno-associated virus and injected into his left thigh; the Koran verse was encoded into DNA but not merged with a virus and was then injected into his right thigh).
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Researchers at Flinders University knocked out a gene known as RCAN1 in mice, hypothesizing that this would increase "non-shivering thermogenesis," which "expends calories as heat rather than storing them as fat" -- the mice were fed a high-calorie diet and did not gain weight.
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Remember when they caught the Golden State Killer by comparing DNA crime-scene evidence to big commercial genomic databases (like those maintained by Ancestry.com, 23 and Me, etc) to find his family members and then track him down?
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George Church's Harvard lab is one of the most celebrated fonts of innovation in the world of life sciences. George's earliest work on the Human Genome Project arguably pre-dated the actual start of that project. Subsequently, he's been involved in the creation of almost a hundred companies - 22 of which he co-founded.
Much of George's most recent and celebrated work has been with a transformationally powerful gene-editing technique called CRISPR, which he co-invented. George and I discuss CRISPR and its jarring ramifications throughout this week's edition of the After on Podcast. You can listen to our interview by searching "After On" in your favorite podcast app, or by clicking right here:
Our conversation begins with a higher-level survey of the field -- one which cleanly and clearly defines CRISPR by placing it into a broader, and also a quite fascinating framework. We cover four topics, which I'll now define up-front for you, so as to make the interview more accessible.
We begin by discussing genetic sequencing. "Sequencing" is a fancy (and rather cool way) of saying, "reading." Your genome is about three billion characters long. It's written in a limited alphabet, of just four letters: A, G, C, and T. And if someone sequences your genome, it simply means they've read it. They haven't modified it in any way. They haven't have cloned you. They've just gotten a readout (kind of like determining your blood type -- only a few billions times more complicated).
George and I next discuss gene editing. Read the rest
This is Ata, a bizarre, tiny mummified skeleton found in a deserted mining town in Chile's Atacama Desert in 2003.
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Cornell archaeobotanist Natalie Mueller harvests "weeds" from across North America, seeking the remnants of "lost crops," the plants cultivated by the people who lived here 2,000 years ago.
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Xinjiang province is the site of intense surveillance and oppression, even by Chinese standards; it's home to the largely Muslim Uyghur minority, and a combination of racism and Islamaphobia drive a uniquely intrusive grade of policing and surveillance.
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Today at the Usenix Security conference, a group of University of Washington researchers will present a paper showing how they wrote a piece of malware that attacks common gene-sequencing devices and encoded it into a strand of DNA: gene sequencers that read the malware are corrupted by it, giving control to the attackers. Read the rest
Wired asked Biologist Neville Sanjana to explain CRISPR (clustered regularly interspaced short palindromic repeats) gene editing to a 7 year-old, a 14 year-old, a college student, a grad student and a CRISPR expert. Read the rest
Last December, I published my review of Andrew "bunnie" Huang's astoundingly great book The Hardware Hacker: Adventures in Making and Breaking Hardware -- without realizing that the book's release had been delayed because the published decided to do some very fancy and cool stuff with the printing process. Read the rest
I've been writing about genius hardware hackers Andrew "bunnie" Huang since 2003, when MIT hung him out to dry
over his book explaining how he hacked the original Xbox; the book
he wrote about that hack has become a significant engineering classic, and his own life has taken a thousand odd turns that we've chronicled here as he's founded companies, hacked hardware, become a China manufacturing guru, and sued the US government
over the anti-hacking provisions of the DMCA.
Alexander McQueen's first collection after graduating from Central Saint Martins was Jack the Ripper Stalks His Victims which included locks of his hair; for her own grad project, called "Pure Human," Central Saint Martins student Tina Gorjanc created a line of clothes and accessories that asks the audience to imagine that it was made from pelts cloned from DNA retrieved from McQueen's hair strands. Read the rest
How can a mild-mannered grasshopper turn into a ferocious locust? Why are humans humans when we have share 80 percent of the same genetic material with a cow? In a fascinating long read at Aeon, David Dobbs delves into the differences between genetic change (evolution as you probably learned it in school) and genetic expression (the amazing powers of natural selection that scientists are only now starting to really understand). Read the rest
At what point does interesting-but-potentially-incorrect-or-misleading information become a potential threat to health? How do you regulate a product that current regulations were never set up to handle? The University of Michigan's Risk Science Center put together this quick cartoon that neatly summarizes the problems and questions at the heart of the FDA's crackdown on 23andMe, which Xeni wrote about on Monday.
A couple of other smart takes on this that have come out in the past couple of days:
• Genomics expert Michael Eisen delves deeper into the question of how we should regulate personal genetic testing.
• Journalist David Dobbs rounded up some diverse opinions. You should pay attention to his blog. He's been doing a lot of great reporting on genetics and culture and is planning on publishing a longer piece on the 23andMe stuff later this week. Read the rest
It's a crazy world out there. IKEA meatballs — which should, ostensibly, be 100% cow — are, in fact, at least partly horse. Meanwhile cow genomes are even more mixed up, containing 25% snake DNA. How the hell does that happen? The venerable Ed Yong explains. Read the rest
If one of your loved ones was sick, what would you be willing to do to help? For millions of Americans in the US, even though they would give up anything and everything, there is no way for them to help. For many rare diseases, because they are so rare, there are no therapies or even diagnostics. What is needed is research. However, very few people have the funding and the access to the technology needed to perform such a study. Non-profits and foundations have been a major force in pushing forward research, but for many rare diseases, no such groups exist. Rare Genomics Institute (RGI) is hoping to change that, especially for children such as Maya.
Struggling with global developmental delays, Maya has not been able to speak, has had problems hearing, and has undergone many surgeries. However, after many genetic tests, there still was no answer. Then Maya’s mother found out about RGI and how they are helping children with rare diseases. With RGI’s help, Maya’s mother was able to connect with researchers and design a custom research project just for her daughter.
Using RGI’s crowdfunding platform, Maya’s family sought to raise the amount needed. The response was overwhelming. Within 6 hours, donors from all over the US gave to their cause in small amounts of $5 - $50 to raise the funds necessary for whole exome sequencing. With the funding available, the scientists were able to start the research study.
In less than a year, there was a promising result. Read the rest