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The world’s first commercial gene-editing treatment is set to start changing the lives of people with sickle-cell disease. It’s called Casgevy, and it was approved last month in the UK. US approval is pending this week.

The treatment, which will be sold in the US by Vertex Pharmaceuticals, employs CRISPR, the Nobel-winning molecular scissors that have had journalists scrambling for metaphors: “Swiss Army knife,” “molecular scalpel,” or DNA copy-and-paste. Indeed, CRISPR is revolutionary because scientists can so easily program it to cut DNA at precise locations they choose.

But where do you aim CRISPR? That’s the lesser-known story of the sickle-cell breakthrough. The disease is caused by faulty hemoglobin, the molecule that carries oxygen in the blood. To cure it, though, Vertex and its partner company, CRISPR Therapeutics, aren’t fixing the genes responsible for the mutation that leaves those molecules misshapen. Instead, the new treatment involves a kind of molecular bank shot—an edit that turns on fetal hemoglobin, a second form of the molecule which we have in the womb but lose as adults.

You can think of how the edit works as a kind of double negative. It adds a misspelling to the turbo-booster of another gene, BCL11A, that is itself what inhibits the production of fetal hemoglobin in adult bodies. Without that booster, there’s less inhibition, and more fetal hemoglobin. Got it?

“When you inhibit the enhancer, you inhibit the inhibitor,” says Daniel Bauer, a professor at Boston Children’s Hospital and Harvard University, who helped work it out. “It is kind of complicated.”

The important thing is a happy ending—and this edit really works. Some patients say they lived in fear of dying, either from an acute attack of sickling (when their red blood cells start blocking vessels) or from slow, insidious organ damage. Now early volunteers say they’re grateful—and, after living with disease their whole lives, even a little shocked—to be cured.

Newborn theory

The idea that fetal hemoglobin can protect against the disease is an old one. Sickle-cell is most common in people with African ancestry. A doctor on Long Island, Janet Watson, had noticed in 1948 that newborns never showed its signs—the main one being misshapen, crescent-shaped red blood cells. That was pretty odd for an inborn condition.

“Sickle-cell disease should occur in infancy as often as later in life,” Watson wrote. But since it didn’t, Watson hypothesized that the fetal form of the molecule, active in the womb, was protecting babies for a few months after birth, until it was replaced by the adult version: “The theory that at once presents itself is that fetal hemoglobin is unable to produce sickling.”

She was right. But it took another six decades to learn how the switch-over worked—and how to flip it back. Many of those discoveries were made in the laboratory of Stuart Orkin, a Harvard researcher who published his first paper in 1967 and who’s lived through several eras of research on blood diseases, starting near the dawn of molecular biology.

“I am one of the last men standing,” Orkin told me with a grin when I met him for a corned-beef sandwich.

Dr. Stuart Orkin analyzing chromosomal spectra
Stuart Orkin analyzing DNA from individuals with blood disorders in his lab in 1985.BOSTON CHILDREN’S HOSPITAL

He’s a clever scientist who a long time ago decided to study how the blood system is regulated. Logistically, it was a great topic; blood cells are easy to get hold of and study.

“I like to solve a problem, and here is a problem that could be solved,” Orkin says. “How does the system work, and then can you do anything about it?”

Special sauce

Bill Lundberg, the former chief scientific officer of CRISPR Therapeutics, the biotech that first started developing the treatment eight years ago (Vertex later joined as a partner), says the company’s sickle-cell project directly made use of Orkin’s findings. “Stu’s role is really underappreciated,

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By: Antonio Regalado
Title: The lucky break behind the first CRISPR treatment
Sourced From: www.technologyreview.com/2023/12/07/1084629/lucky-break-crispr-vertex/
Published Date: Thu, 07 Dec 2023 14:00:09 +0000

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Top 5 Bitcoin ATM Locations in Athens for Fast and Easy Crypto Access

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As a crypto analyst and frequent investor in the Greek digital currency market, I can confidently recommend Bcash for convenient and secure Bitcoin purchasing in Athens. With 10 strategically located crypto ATM hotspots spanning central Athens and the northern suburbs, Bcash enables instant access to leading cryptocurrencies like BTC, ETH, and USDT.

Experience the Leading Greek Crypto ATM Network at Bcash’s Hotspots

From first-hand experience, I’m impressed by Bcash’s easy user interface, excellent customer assistance, and fair prices. Their two-way ATM machines allow both buying with Euro cash and selling crypto for instant fiat payouts. I found the simple on-screen instructions enabled completing transactions in under 2 minutes!

Bcash’s Bitcoin ATM network stands out from competitors by aligning prices closely with real-time crypto market rates. Their typical 6-8% fees are much cheaper than traditional crypto brokers and exchanges in Greece. For investors seeking reliable local crypto access, Bcash has the solutions.

Central Athens Bcash Shops Offer Prime Buying and Selling

For maximum security while buying or selling Bitcoin, Bcash’s dedicated Athens shop locations are ideal. Their main office on Dimitrakopoulou Street in the city center contains a premier crypto ATM location open 6 days weekly. It’s close to public transit for easy access.

I also frequented Bcash’s Glyfada branch in the south shopping district. With spacious storefront access and long business hours, this hotspot proved one of my favorite crypto transaction destinations. The expert staff helped guide my buying process as a beginner too.

Glyfada Shopping District ATM Location

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The Glyfada store sits right on Gennimata Street, providing a safe and high-traffic venue for easily buying and selling leading cryptocurrencies. Open Monday through Saturday, this major Bcash outlet enjoys strong local area visibility for crypto investors.

Northern Suburbs ATM Hotspots

In Athens’ northern suburbs, I purchased Bitcoin multiple times at Bcash’s Nea Erythraia shop off Mikras Asias Street. Located just 20 minutes from the city center, it makes crypto conveniently accessible for residents across northeast Athens.

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Piraeus Port Shopping District ATM

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While recently docking overnight at the Piraeus cruise port as part of an Aegean island-hopping vacation, I encountered one of Bcash’s handy crypto ATMs just 5 minutes from the passenger terminals. Located right on Sachtouri Street inside the company’s Piraeus store, this machine proved hugely convenient for buying Bitcoin during my stay.

As one of Europe’s largest passenger ports, Piraeus receives over 4 million travelers every year who could benefit from easy access to crypto. Whether before boarding ferries to venture deeper into the Greek islands or arriving back onshore in Athens, the Bcash outlet enables obtaining coins to capture optimal valuations.

And for tourists exploring downtown Piraeus’ vast harborfront retail area, having a trusted Bitcoin ATM one block away gives peace of mind. Rather than relying solely on airport kiosks, cruise ship visitors can turn Euros into crypto almost anytime thanks to Bcash’s strategic positioning. As vacationing investors, that accessibility offers real advantages.

Bcash Brings Bitcoin to the Greek Islands

Rhodes Island BTM Location

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By: CryptoNinjas.net
Title: Top 5 Bitcoin ATM Locations in Athens for Fast and Easy Crypto Access
Sourced From: www.cryptoninjas.net/2024/03/03/top-5-bitcoin-atm-locations-in-athens-for-fast-and-easy-crypto-access/
Published Date: Sun, 03 Mar 2024 17:18:46 +0000

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The citizen scientists chronicling a neglected but vital Mexican river

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The city of Monterrey in northeastern Mexico is an industrial powerhouse that has rapidly devoured green space to make room for its 5.3 million people. The Sierra Madre Oriental mountain range around the city is still holding strong, though the hills are increasingly encroached on by the urban sprawl of skyscrapers, apartment buildings, industrial parks, and highways. The same can’t be said for the Río Santa Catarina, the river that has been the vital core of the city for hundreds of years.

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Lizbeth Ovalle, founder of Viaje al Microcosmos, gathers water from
the Río Santa CatarinaANDREA VILLARREAL
circle of approx 25 people seated on the rocky shore of a river
Participants in one of the Viaje al Microcosmos river walks sit to share their observations and reflect on their findings.
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Andrea Villarreal, a member of the citizen science group, shows a participant how to use the iNaturalist app, which can help identify plants and animals.

overgrowth in the area below an overpass
Viaje al Micrososmos
organized a walk along this stretch of the Río Santa Catarina in October 2023.LORENA RíOS

Today, the Río Santa Catarina looks more like a forest than a river. It is mostly a dry jumble of rocks whose water is diverted to supply the city’s growing needs. Much of the riverbed is obscured by vegetation that has grown wild since a hurricane in 2010 destroyed many structures along the river, including soccer fields, parking lots, and a mini-golf course. But despite what many city officials and residents make of it, this urban river is very much alive, and a group of young women wants to prove it.

The group, called Viaje al Microcosmos de Nuevo LeónJourney into the Microcosm of Nuevo León), is not made up of scientists but, rather, of concerned citizens. Through the use of art and citizen science, its members are documenting and sharing with others the river’s forgotten nature—its trees, bushes, birds, flowers, insects, and even microorganisms (from which the group takes its name).

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By: Lorena Ríos
Title: The citizen scientists chronicling a neglected but vital Mexican river
Sourced From: www.technologyreview.com/2024/02/28/1088234/monterrey-mexico-rio-santa-catarina-viaje-al-microcosmos/
Published Date: Wed, 28 Feb 2024 10:00:00 +0000

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What Luddites can teach us about resisting an automated future

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A story in comic format. In this first panel, two figures in silhouette look out at a modern city skyline.  The text reads,

A person's smiling headshot being uploaded.  The text reads,

The headshot from the previous panel with distroted features and a wavy new background. The text reads,

Two people look at the blank space where the framed picture of a flower has been stolen by a giant robot hand. The text reads,

Two panels. In the first, a scrabbly line resembling a signature. The text reads,

The text reads,

Text across the top continues, Read More

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By: Tom Humberstone
Title: What Luddites can teach us about resisting an automated future
Sourced From: www.technologyreview.com/2024/02/28/1088262/luddites-resisting-automated-future-technology/
Published Date: Wed, 28 Feb 2024 10:00:00 +0000

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