UK first to approve CRISPR treatment for human diseases


The landmark decision could transform the treatment of sickle-cell disease and β-thalassaemia — but the technology is expensive.


In a world first, the UK medicines regulator has approved a therapy that uses the CRISPR–Cas9 gene-editing tool as a treatment. The decision marks another high point for a biotechnology that has been lauded as revolutionary in the decade since its discovery.


The therapy, called Casgevy, will treat the blood conditions sickle-cell disease and β-thalassaemia. Sickle-cell disease, also known as sickle-cell anaemia, can cause debilitating pain, and people with β-thalassaemia often require regular blood transfusions.


“This is a landmark approval which opens the door for further applications of CRISPR therapies in the future for the potential cure of many genetic diseases,” said Kay Davies, a geneticist at the University of Oxford, UK, in comments to the UK Science Media Centre (SMC).


Nature magazine explains the research behind the treatment and explores what’s next.

What research led to the approval?

The approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows promising results from clinical trials that tested a one-time treatment, which is administered by intravenous infusion. The therapy was developed by the pharmaceutical company Vertex Pharmaceuticals in Boston, Massachusetts, and biotechnology company CRISPR Therapeutics in Zug, Switzerland.


The trial for sickle-cell disease has followed 29 out of 45 participants long enough to draw interim results. Casgevy completely relieved 28 of those people of debilitating episodes of pain for at least one year after treatment. Researchers also tested the treatment for a severe form of β-thalassaemia, which is conventionally treated with blood transfusions roughly once a month. In this trial, 54 people received Casgevy, of which 42 participated for long enough to provide interim results. Among those 42 participants, 39 did not need a red-blood-cell transfusion for at least one year. The remaining three had their need for blood transfusions reduced by more than a 70%.

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The GPT to rule them all: ‘ScienceGPT’ is being trained from data from the Aurora supercomputer

Scientists are training a gargantuan one-trillion-parameter generative AI system dubbed ‘ScienceGPT’ based on scientific data from the newly established Aurora supercomputer.


The AuroraGPT AI model, which is being trained by researchers at the Argonne National Lab (ALN) in Illinois, USA, is powered by Intel‘s Ponte Vecchio GPUs which provide the main computing power, and is being backed by the US government. Training could take months to complete, according to HPC Wire, with training currently limited to 256 of the roughly 10,000 nodes of the Aurora supercomputer, before this is scaled up over time. Even given this limitation, Intel and ANL are only testing the model training on a string of 64 nodes, with caution due to Aurora’s unique design as a supercomputer.


At one trillion parameters, ScienceGPT will be one of the largest LLMs out there. While it won’t quite hit the size of the reported 1.7-trillion-parameter GPT-4, developed by OpenAI, it’ll be almost twice as large as the 560-billion-parameter Pathways Language Model, which powers Google‘s Bard. “It combines all the text, codes, specific scientific results, papers, into the model that science can use to speed up research,” said Ogi Brkic, vice president and general manager for data center and HPC solutions, in a press briefing. It’ll operate like ChatGPT, but it’s yet unclear at the moment whether it will be multimodal, in that it will generate different kinds of media like text, images, and video. 



Aurora  – which will be the second exascale supercomputer in US history – has just established itself on the Top500 list of the most powerful supercomputers after years of being developed.  It’s the second-most powerful supercomputer after Froniter, and is powered by 60,000 Intel GPUs while boating 10,000 computing nodes over 166 racks, alongside more than 80,000 networking nodes.  It is still being finished, however, and will likely exceed Frontier’s performance when it’s fully up to speed, and all testing and finetuning is complete, said Top500.

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Neuroscientists Re-create Pink Floyd Song from Listeners’ Brain Activity


For the first time, scientists have demonstrated that the brain’s electrical activity can be decoded and used to reconstruct music.
Artificial intelligence has turned the brain’s electrical signals into somewhat garbled classic rock”

“Neuroscientists have reconstructed recognizable audio of a 1979 Pink Floyd song by using machine learning to decode electrical activity in the brains of listeners. As study participants undergoing surgery listened to “Another Brick in the Wall (Part 1),” electrodes placed on the surface of the brain captured the activity of regions attuned to the song’s acoustic profile. “


Neuroscientists have worked for decades to decode what people are seeing, hearing or thinking from brain activity alone. In 2012 a team that included the new study’s senior author—cognitive neuroscientist Robert Knight of the University of California, Berkeley—became the first to successfully reconstruct audio recordings of words participants heard while wearing implanted electrodes. Others have since used similar techniques to reproduce recently viewed or imagined pictures from participants’ brain scans, including human faces and landscape photographs. But the recent PLOS Biology paper by Knight and his colleagues is the first to suggest that scientists can eavesdrop on the brain to synthesize music.


“These exciting findings build on previous work to reconstruct plain speech from brain activity,” says Shailee Jain, a neuroscientist at the University of California, San Francisco, who was not involved in the new study. “Now we’re able to really dig into the brain to unearth the sustenance of sound.”


To turn brain activity data into musical sound in the study, the researchers trained an artificial intelligence model to decipher data captured from thousands of electrodes that were attached to the participants as they listened to the Pink Floyd song while undergoing surgery. Why did the team choose Pink Floyd—and specifically “Another Brick in the Wall (Part 1),”? “The scientific reason, which we mention in the paper, is that the song is very layered. It brings in complex chords, different instruments and diverse rhythms that make it interesting to analyze,” says Ludovic Bellier, a cognitive neuroscientist and the study’s lead author. “The less scientific reason might be that we just really like Pink Floyd.”

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Unlocking the secrets of spin with high-harmonic probes (Heusler compound)


Deep within every piece of magnetic material, electrons dance to the invisible tune of quantum mechanics. Their spins, akin to tiny atomic tops, dictate the magnetic behavior of the material they inhabit. This microscopic ballet is the cornerstone of magnetic phenomena, and it’s these spins that a team of JILA researchers—headed by JILA Fellows and University of Colorado Boulder professors Margaret Murnane and Henry Kapteyn—has learned to control with remarkable precision, potentially redefining the future of electronics and data storage.



In a Science Advances publication, the JILA team—along with collaborators from universities in Sweden, Greece, and Germany—probed the spin dynamics within a special material known as a Heusler compound: a mixture of metals that behaves like a single magnetic material.


For this study, the researchers utilized a compound of cobalt, manganese, and gallium, which behaved as a conductor for electrons whose spins were aligned upwards and as an insulator for electrons whose spins were aligned downwards. Using a form of light called extreme ultraviolet high-harmonic generation (EUV HHG) as a probe, the researchers could track the re-orientations of the spins inside the compound after exciting it with a femtosecond laser, which caused the sample to change its magnetic properties. The key to accurately interpreting the spin re-orientations was the ability to tune the color of the EUV HHG probe light.


“In the past, people haven’t done this color tuning of HHG,” explained co-first author and JILA graduate student Sinéad Ryan. “Usually, scientists only measured the signal at a few different colors, maybe one or two per magnetic element at most.” In a monumental first, the JILA team tuned their EUV HHG light probe across the magnetic resonances of each element within the compound to track the spin changes with a precision down to femtoseconds (a quadrillionth of a second).


“On top of that, we also changed the laser excitation fluence, so we were changing how much power we used to manipulate the spins,” Ryan elaborated, highlighting that that step was also an experimental first for this type of research.

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New antifungal molecule kills fungi without toxicity to human and murine cells


Terrible to terrific: A new antifungal molecule tweaks a powerful drug to harness its power against infection while doing away with its toxicity.


A new antifungal molecule, devised by tweaking the structure of prominent antifungal drug Amphotericin B, has the potential to harness the drug’s power against fungal infections while doing away with its toxicity, researchers at the University of Illinois Urbana-Champaign and collaborators at the University of Wisconsin-Madison report in the journal Nature.


Amphotericin B, a naturally occurring small molecule produced by bacteria, is a drug used as a last resort to treat fungal infections. While AmB excels at killing fungi, it is reserved as a last line of defense because it also is toxic to the human patient – particularly the kidneys. 


“Fungal infections are a public health crisis that is only getting worse. And they have the potential, unfortunately, of breaking out and having an exponential impact, kind of like COVID-19 did. So let’s take one of the powerful tools that nature developed to combat fungi and turn it into a powerful ally,” said research leader Dr. Martin D. Burke, an Illinois professor of chemistry, a professor in the Carle Illinois College of Medicine and also a medical doctor. 


“This work is a demonstration that, by going deep into the fundamental science, you can take a billion-year head start from nature and turn it into something that hopefully is going to have a big impact on human health,” Burke said. 



Burke’s group has spent years exploring AmB in hopes of making a derivative that can kill fungi without harm to humans. In previous studies, they developed and leveraged a building block-based approach to molecular synthesis and teamed up with a group specializing in molecular imaging tools called solid-state nuclear magnetic resonance, led by professor Chad Rienstra at the University of Wisconsin-Madison. Together, the teams uncovered the mechanism of the drug: AmB kills fungi by acting like a sponge to extract ergosterol from fungal cells. 


In the recent work, Burke’s group worked again with Rienstra’s group to find that AmB similarly kills human kidney cells by extracting cholesterol, the most common sterol in people. The researchers also resolved the atomic-level structure of AmB sponges when bound to both ergosterol and to cholesterol. 


“The atomic resolution models were really the key to zoom in and identify these very subtle differences in binding interactions between AmB and each of these sterols,” said Illinois graduate student Corinne Soutar, a co-first author of the paper. “Using this structural information along with functional and computational studies, we achieved a significant breakthrough in understanding how AmB functions as a potent fungicidal drug,” Rienstra said. “This provided the insights to modify AmB and tune its binding properties, reducing its interaction with cholesterol and thereby reducing the toxicity.” 

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If You Had Gamma Ray Eyes the Moon Would Glow Brighter Than the Sun


If our eyes could see high-energy radiation called gamma rays, the Moon would appear brighter than the Sun! That’s how NASA’s Fermi Gamma-ray Space Telescope has seen our neighbor in space for the past decade. Gamma-ray observations are not sensitive enough to clearly see the shape of the Moon’s disk or any surface features. Instead, Fermi’s Large Area Telescope (LAT) detects a prominent glow centered on the Moon’s position in the sky.


Mario Nicola Mazziotta and Francesco Loparco, both at Italy’s National Institute of Nuclear Physics in Bari, have been analyzing the Moon’s gamma-ray glow as a way of better understanding another type of radiation from space: fast-moving particles called cosmic rays. “Cosmic rays are mostly protons accelerated by some of the most energetic phenomena in the universe, like the blast waves of exploding stars and jets produced when matter falls into black holes,” explained Mazziotta.


Because the particles are electrically charged, they’re strongly affected by magnetic fields, which the Moon lacks. As a result, even low-energy cosmic rays can reach the surface, turning the Moon into a handy space-based particle detector. When cosmic rays strike, they interact with the powdery surface of the Moon, called the regolith, to produce gamma-ray emission. The Moon absorbs most of these gamma rays, but some of them escape.


Mazziotta and Loparco analyzed Fermi LAT lunar observations to show how the view has improved during the mission. They rounded up data for gamma rays with energies above 31 million electron volts — more than 10 million times greater than the energy of visible light — and organized them over time, showing how longer exposures improve the view.


“Seen at these energies, the Moon would never go through its monthly cycle of phases and would always look full,” said Loparco. As NASA sets its sights on sending humans to the Moon by 2024 through the Artemis program, with the eventual goal of sending astronauts to Mars, understanding various aspects of the lunar environment take on new importance. These gamma-ray observations are a reminder that astronauts on the Moon will require protection from the same cosmic rays that produce this high-energy gamma radiation.


While the Moon’s gamma-ray glow is surprising and impressive, the Sun does shine brighter in gamma rays with energies higher than 1 billion electron volts. Cosmic rays with lower energies do not reach the Sun because its powerful magnetic field screens them out. But much more energetic cosmic rays can penetrate this magnetic shield and strike the Sun’s denser atmosphere, producing gamma rays that can reach Fermi.


Although the gamma-ray Moon doesn’t show a monthly cycle of phases, its brightness does change over time. Fermi LAT data show that the Moon’s brightness varies by about 20% over the Sun’s 11-year activity cycle. Variations in the intensity of the Sun’s magnetic field during the cycle change the rate of cosmic rays reaching the Moon, altering the production of gamma rays.


Download the graphic and related multimedia in HD formats from NASA Goddard’s Scientific Visualization Studio

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First-Ever Observation of a Virus Attaching to Another Virus


No one had ever seen one virus latching onto another virus, until anomalous sequencing results sent a UMBC team down a rabbit hole leading to a first-of-its-kind discovery. It’s known that some viruses, called satellites, depend not only on their host organism to complete their life cycle, but also on another virus, known as a “helper,” explains Ivan Erill, professor of biological sciences.


The satellite virus needs the helper either to build its capsid, a protective shell that encloses the virus’s genetic material, or to help it replicate its DNA. These viral relationships require the satellite and the helper to be in proximity to each other at least temporarily, but there were no known cases of a satellite actually attaching itself to a helper—until now.


In a paper published in The ISME Journal, a UMBC team and colleagues from Washington University in St. Louis (WashU) describe the first observation of a satellite bacteriophage (a virus that infects bacterial cells) consistently attaching to a helper bacteriophage at its “neck”—where the capsid joins the tail of the virus. In detailed electron microscopy images taken by Tagide deCarvalho, assistant director of the College of Natural and Mathematical Sciences Core Facilities and first author on the new paper, 80 percent (40 out of 50) helpers had a satellite bound at the neck. Some of those that did not had remnant satellite tendrils present at the neck. Erill, senior author on the paper, describes them as appearing like “bite marks.” “When I saw it, I was like, I can’t believe this,” deCarvalho says. “No one has ever seen a bacteriophage—or any other virus—attach to another virus.”


Research is published in ISME (October 31, 2023): 

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Google Maps just got a big AI update – here are the 5 best new features

Google has been busy injecting AI-powered features into all of its services this year, and some of the most promising are in Google Maps. Well, this week those features – including the long-awaited Immersive View for Routes – are going to start rolling out on your Android or iOS phone, alongside some new tricks.

The biggest news is the rollout of Immersive View for Routes in 15 cities, starting this week. The feature combines Street view, aerial imagery, and live information like weather and traffic to give you an aerial, photo-realistic preview of your planned Google Maps route – so we’re excited to take it for a spin soon.

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