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2025-12-03 05:29:25

Ollie Chu was three years old when he received an infusion that would change his life.

Born with a rare inherited condition called Hunter syndrome, Ollie’s body couldn’t produce an enzyme that breaks down complex sugars.

Just a few months after his birth, the sugars had built up everywhere, wreaking havoc on lungs, liver, skin, and brain. In Hunter syndrome, joints stiffen and airways narrow, making it hard to breathe. The brain also struggles to grow, resulting in developmental delays and cognitive problems. Most kids diagnosed with the condition don’t live past 20.

There are a few treatments. One drug on the market counteracts some bodily symptoms but at a hefty price. It must be taken weekly for life and can’t rescue the brain. Another option is a full bone marrow replacement. While this offers a long-term solution, the procedure is risky for toddlers and depends on the availability of matching donors, who are few and far between.

Ollie’s treatment is new. Roughly a year ago, researchers at the University of Manchester removed stem cells from his body, genetically inserted a functional copy of the gene encoding the missing enzyme, and infused the edited cells back into his body through a catheter.

Now, he no longer depends on weekly drug infusions. “[He] is doing great since having the gene therapy. We have seen dramatic improvements, and he continues to grow physically and cognitively,” said his dad, Ricky, in a press release.

Ollie is one of five very young children in an ongoing clinical trial of gene therapy for Hunter syndrome. Led by the Royal Manchester Children’s Hospital and collaborators, researchers hope the one-and-done therapy will slash treatment time and offer a lasting solution.

“Gene therapy is not only safer and more effective [than bone marrow transplant], but it enables us to use the child’s own cells which cuts out the need to find a donor,” said joint clinical lead Rob Wynn. If successful, the principles could be adapted for other genetic diseases.

Broken Waste Plant

Cells are constantly building, destroying, and recycling proteins. They monitor the levels of different molecules—sugars, fats, and proteins—and shuttle excess to the lysosome.

Think of the lysosome as a cell’s “stomach.” Each bubble-like structure contains acidic fluids and a menagerie of enzymes to break down different types of molecules.

One of these enzymes, called iduronate-2-sulfatase (IDS), is missing in Hunter syndrome. The enzyme exists in all cells, but it’s most active in the liver, skin, immune system, and brain. Rather than staying put, IDS loves to roam about and explore neighboring cells. In other words, if only a fraction of cells can make the enzyme, its effects would still spread beyond just the treated ones.

The enzyme replacement therapy Ollie and other kids with Hunter syndrome begin early in life relies on IDS. Here, the enzyme is infused into the bloodstream where it’s absorbed into multiple tissues to help clean out toxic sugars. The treatment improves lung and liver function and helps with joint mobility. But due to its large size, it can’t enter the brain. Hence, the disease continues to attack neural function.

At the root of Hunter syndrome is the gene that produces IDS. Using viruses and gene editing, studies have shown that delivering a healthy version of the gene to mice boosts production of the enzyme. Some genetic diseases have only a single DNA letter change. But the IDS gene mutates in hundreds of ways, making it difficult to engineer a universal gene therapy.

A bone marrow transplant from a matching healthy donor is one workaround. Donor stem cells gradually develop into a range of healthy blood and immune cells. Because they have a normal version of the IDS gene, these cells pump the missing enzyme throughout the body.

A transplant is a one-and-done treatment, but the recipient must take immunosuppressant drugs for the rest of their life, increasing the chance of infections. And the wait for a matching donor can be very long.

Complete Replacement

In Ollie’s treatment, researchers harvested his own stem cells for gene therapy. Because the cells come from his body, they’re more likely to evade immune rejection.

The approach is based on a mouse study by Brian Bigger and colleagues, who is also co-leading the clinical trial. It uses a viral carrier, stripped of disease-causing genes, to shuttle a healthy IDS gene into blood stem cells outside the body. The edited cells are then infused back into the patient. The virus inserts the gene directly into the cell’s genome, ensuring the replacement isn’t lost when the cells divide.

Rather than using a natural version of IDS, the team added a snippet to the gene that helps the enzyme better tunnel into the brain. Once infused, the edited stem cells multiply into a variety of blood and immune cells that roam the body and release the working enzyme.

In mice modeling Hunter syndrome, a single treatment completely reversed brain symptoms for up to 16 months—or almost their entire lifespan. Other organs also benefited without notable side effects.

In late 2024, Ollie, at just three years of age, underwent a similar procedure. His doctors collected and isolated his blood stem cells and genetically tweaked them to churn out the missing enzyme. As he watched cartoons, the team infused two doses of the edited cells through a catheter. He quickly recovered and was discharged from the hospital a few days later.

Within three months of the infusion, Ollie was able to come off the weekly drug infusions that had dominated his life. His speech and motor abilities improved, allowing him to ride a tricycle, hang out with friends, and enjoy a normal childhood.

“I want to pinch myself every time I tell people that Oliver is making his own enzymes,” his mother Jingru told the BBC. “Every time we talk about it I want to cry because it’s just so amazing.”

The team is recruiting other children with Hunter syndrome in the ongoing clinical trial to further test safety and efficacy. Because symptoms progress so rapidly before causing brain damage, the trial only accepts patients between three and 12 months of age. (At first, doctors thought Ollie was too old, but testing showed his condition had progressed only a little.) Once treated, the children will be followed for two years to gauge the therapy’s effects against common symptoms, such as delayed learning, hearing issues, and heart and lung problems.

If successful, the same gene-editing approach could be used to treat other inherited diseases involving stem cells. Ollie’s parents are hopeful the therapy might be extended to older children, including his five-year-old brother Skyler, who also has Hunter syndrome but is currently too old for the trial.

Still, to his father Ricky, the experimental treatment has been a success.

“We’re excited for Ollie’s future. Seeing the difference for Ollie pre-and post-transplant has made us believers,” he said. “We hope that one day, a treatment becomes available for all children at all stages of Hunter syndrome.”

The post Groundbreaking Gene Therapy Transforms Life of Boy With Devastating Disorder appeared first on SingularityHub.

2025-12-02 04:52:51

Space is getting busier as national space agencies and private companies increase the tempo of launches. But today’s approach to space exploration is unsustainable, say researchers, and we need to do more to make sure the orbital economy is a circular one.

While companies like SpaceX have made progress with reusable rockets, most launch vehicles are used only once, and their remains are left to either burn up in the atmosphere or clutter low-Earth orbit. They also dump huge quantities of greenhouse gases and ozone-depleting chemicals into the upper atmosphere.

Satellites are similarly unsustainable. After completing missions, they’re often either moved to a “graveyard orbit,” or worse, they add to the growing pile of space junk making low-Earth orbit increasingly hard to navigate.

As the pace of launches grows, these approaches are no longer viable, say researchers. In a paper published in Chem Circularity, scientists argue we need to shift to a “circular space economy” designed around the principles of reducing, repairing, and recycling.

“As space activity accelerates, from mega-constellations of satellites to future lunar and Mars missions, we must make sure exploration doesn’t repeat the mistakes made on Earth,” the University of Surrey’s Jin Xuan says in a press release. “A truly sustainable space future starts with technologies, materials, and systems working together.”

Progress already made in shifting industries like electronics and automotive manufacturing to more circular practices could provide a template for the space economy, say the researchers.

To reduce waste in the industry, they say spacecraft need to be more durable to increase their lifespans. This could slash material waste from the vehicles themselves and reduce the number of launches required.

Making spacecraft more repairable could also play an important role, they note. To make this possible, space stations would need to become hubs that carry out maintenance and build spacecraft components. They could also refuel satellites already in orbit to extend their lifespans.

Recycling spacecraft is more challenging due to the enormous amount of wear and tear they undergo in the harsh conditions of space and the punishing process of re-entering the atmosphere. The researchers say companies need to further develop soft-landing systems like parachutes and airbags to ensure vehicles can be brought back safely.

The study also calls for systematic efforts to clear existing orbital debris. These would reduce the risk of collisions but could also recover valuable materials. The work would require new tools like robotic arms and nets that can safely capture spacecraft moving at thousands of miles per hour.

The biggest challenge, the researchers say, is this would represent a fundamental shift in the way the space industry operates. That means piecemeal progress on individual components or processes won’t cut it: What’s needed is a system-wide commitment to radically different ways of operating.

“We need innovation at every level, from materials that can be reused or recycled in orbit and modular spacecraft that can be upgraded instead of discarded, to data systems that track how hardware ages in space,” says Xuan. “But just as importantly, we need international collaboration and policy frameworks to encourage reuse and recovery beyond Earth.”

That may prove challenging in an arena that has been characterized by intense geopolitical competition. But we have a golden opportunity to avoid the same mistakes we have made here on Earth.

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2025-11-30 04:29:19

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2025-11-28 23:00:00

You might think a honey bee foraging in your garden and a browser window running ChatGPT have nothing in common. But recent scientific research has been seriously considering the possibility that either, or both, might be conscious.

There are many different ways of studying consciousness. One of the most common is to measure how an animal—or artificial intelligence—acts.

But two new papers on the possibility of consciousness in animals and AI suggest new theories for how to test this—one that strikes a middle ground between sensationalism and knee-jerk skepticism about whether humans are the only conscious beings on Earth.

A Fierce Debate

Questions around consciousness have long sparked fierce debate.

That’s in part because conscious beings might matter morally in a way that unconscious things don’t. Expanding the sphere of consciousness means expanding our ethical horizons. Even if we can’t be sure something is conscious, we might err on the side of caution by assuming it is—what philosopher Jonathan Birch calls the precautionary principle for sentience.

The recent trend has been one of expansion.

For example, in April 2024 a group of 40 scientists at a conference in New York proposed the New York Declaration on Animal Consciousness. Subsequently signed by over 500 scientists and philosophers, this declaration says consciousness is realistically possible in all vertebrates (including reptiles, amphibians and fishes) as well as many invertebrates, including cephalopods (octopus and squid), crustaceans (crabs and lobsters) and insects.

In parallel with this, the incredible rise of large language models, such as ChatGPT, has raised the serious possibility that machines may be conscious.

Five years ago, a seemingly ironclad test of whether something was conscious was to see if you could have a conversation with it. Philosopher Susan Schneider suggested if we had an AI that convincingly mused on the metaphysics of consciousness, it may well be conscious.

By those standards, today we would be surrounded by conscious machines. Many have gone so far as to apply the precautionary principle here too: the burgeoning field of AI welfare is devoted to figuring out if and when we must care about machines.

Yet all of these arguments depend, in large part, on surface-level behavior. But that behavior can be deceptive. What matters for consciousness is not what you do, but how you do it.

Looking at the Machinery of AI

A new paper in Trends in Cognitive Sciences that one of us (Colin Klein) coauthored, drawing on previous work, looks to the machinery rather than the behavior of AI.

It also draws on the cognitive science tradition to identify a plausible list of indicators of consciousness based on the structure of information processing. This means one can draw up a useful list of indicators of consciousness without having to agree on which of the current cognitive theories of consciousness is correct.

Some indicators (such as the need to resolve trade-offs between competing goals in contextually appropriate ways) are shared by many theories. Most other indicators (such as the presence of informational feedback) are only required by one theory but indicative in others.

Importantly, the useful indicators are all structural. They all have to do with how brains and computers process and combine information.

The verdict? No existing AI system (including ChatGPT) is conscious. The appearance of consciousness in large language models is not achieved in a way that is sufficiently similar to us to warrant attribution of conscious states.

Yet at the same time, there is no bar to AI systems—perhaps ones with a very different architecture to today’s systems—becoming conscious.

The lesson? It’s possible for AI to behave as if conscious without being conscious.

Measuring Consciousness in Insects

Biologists are also turning to mechanisms—how brains work—to recognize consciousness in non-human animals.

In a new paper in Philosophical Transactions B, we propose a neural model for minimal consciousness in insects. This is a model that abstracts away from anatomical detail to focus on the core computations done by simple brains.

Our key insight is to identify the kind of computation our brains perform that gives rise to experience.

This computation solves ancient problems from our evolutionary history that arise from having a mobile, complex body with many senses and conflicting needs.

Importantly, we don’t identify the computation itself—there is science yet to be done. But we show that if you could identify it, you’d have a level playing field to compare humans, invertebrates, and computers.

The Same Lesson

The problem of consciousness in animals and in computers appear to pull in different directions.

For animals, the question is often how to interpret whether ambiguous behavior (like a crab tending its wounds) indicates consciousness.

For computers, we have to decide whether apparently unambiguous behavior (a chatbot musing with you on the purpose of existence) is a true indicator of consciousness or mere roleplay.

Yet as the fields of neuroscience and AI progress, both are converging on the same lesson: when making judgement about whether something is consciousness, how it works is proving more informative than what it does.

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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2025-11-27 23:00:00

The gene editor CRISPR is tackling fatty molecules in the body that contribute to one of the world’s top killers: cardiovascular disease.

At the American Heart Association Scientific Sessions 2025 (AHA 2025) this month, Scribe Therapeutics, a startup based in Alameda, California, presented three CRISPR formulations that slashed dangerously high lipid levels in lab-grown cells, mice, and monkeys.

With a single injection, their flagship formulation lowered “bad cholesterol” levels in primates for over 515 days. The treatment used a type of genetic manipulation called epigenome editing that doesn’t directly change the genetic code, potentially reducing side effects.

Two other CRISPR formulations targeted lipoprotein(a) and triglycerides, both fatty substances that form clumps inside blood vessels when at high levels. An injection in mice slashed the molecules by over 95 percent in early trials.

The therapies join other emerging efforts using CRISPR to tackle cardiovascular disease. If the results translate to humans, a daily pill—often taken for decades—may become a thing of the past.

“These results demonstrate that comprehensive engineering of CRISPR technologies can produce medicines with markedly improved safety and performance, surpassing the limitations of early Cas9-based systems,” Benjamin Oakes, cofounder and CEO of Scribe, said in a press release.

A Hefty Problem

High cholesterol haunts millions of Americans. A silent killer, the fatty molecules clog up blood vessels and raise the risk of heart attack, vascular disease, and stroke. Physicians recommend daily statins and dietary changes to manage cholesterol levels, but the regime is hard to follow—especially for years or decades.

Cholesterol comes in multiple forms. Some of these protect the heart and blood vessels. Others lead to clogged arteries. LDL, or low-density lipoprotein, normally transports molecules from the liver to the body’s cells to maintain essential functions, such as building membranes, producing hormones, and creating vitamin D. Too much LDL, however, leads to a buildup of plaques that harden blood vessels and narrow their diameter. This means the heart must work harder to pump blood through the body.

After years of research, scientists identified a gene called PCSK9 that, if overactive, increases the levels of LDL circulating in the blood. FDA-approved drugs that inhibit the PCSK9 protein show promise for lowering cholesterol. But inhibiting the gene itself could offer a longer-term solution.

There have been early successes. In 2023, a small clinical trial in people genetically prone to dangerously high levels of cholesterol found a single infusion of a precise gene editor decreased artery-clogging fat by almost half. Participants had a single mutated DNA letter in the PCSK9 gene that caused their LDL levels to skyrocket. Using base editing—a version of CRISPR—the team engineered a therapy to correct the genetic typo.

A similar strategy could also benefit other populations with high cholesterol. However, base editing permanently alters the genome and could trigger unexpected DNA changes.

Enter epigenetic editors. Rather than directly altering DNA letters, this technology targets the molecular machinery that switches genes on or off. Because epigenetic editors don’t directly change the genetic code, the approach could potentially be safer than gene editing.

Last year, one team employed designer molecules called zinc-finger proteins, a favorite gene-editing tool predating CRISPR, to shut down PCSK9 without changing the gene itself. A single injection slashed cholesterol levels in mice and kept them low for nearly a year—roughly half the mice’s lifespan.

AHA 2025 built on those results.

Scribe developed an epigenetic silencer to suppress PCSK9 using CRISPR-CasX. Like the original version, CRISPR-Cas9, CRISPR-CasX has a guide RNA that tethers CasX—a tiny scissor enzyme—to genes involved in regulating PCSK9 activity and shuts them down.

In monkeys, a single infusion of the treatment slashed LDL levels up to 68 percent. Unlike DNA edits, epigenetic modifications are often lost when cells divide, meaning the drug could lose efficacy over time, especially in rapidly regenerating organs like the liver. But the monkey’s LDL levels remained low for over 515 days without otherwise stressing their livers. Also, the drug didn’t notably change the activity of other genes in cultured human liver cells, suggesting it’s precise.

The data strengthens “the case for a new class of durable epigenetic medicines for large patient populations,” wrote the company in a press release.

Trio of Trouble

PCSK9 isn’t the only gene involved in heart disease. CRISPR Therapeutics, headquartered in Switzerland, worked with the Cleveland Clinic Foundation to find another gene related to high cholesterol levels: ANGPTL3. Studies show people born with dysfunctional versions of the gene naturally have lower LDL levels and risk of heart disease.

The team used CRISPR-Cas9 to disable the gene and recruited 15 people with various blood lipid diseases to test the treatment’s safety profile. Two weeks after a shot, participants’ ANGPTL3 protein and LDL levels dropped significantly and remained low for at least 60 days. Results from the trial, also presented at AHA 2015, found that the treatment was well tolerated overall.

“This is really unprecedented,” said author Luke J. Laffin in a press briefing. “If confirmed in larger trials, this one-and-done approach could transform care for people with lifelong lipid disorders and dramatically reduce cardiovascular risk.”

Artery-blocking lipids beyond LDL are now also in CRISPR’s crosshairs.

Lipoprotein(a) is a mysterious nanoball of fat that’s somewhat similar to LDL in structure but with a more complex mix of components. The substance deposits cholesterol as it roams blood vessels—including smaller ones involved in healing and regeneration. An estimated 30 percent of people worldwide have abnormally high levels of lipoprotein(a). This is mainly due to genetic risks and is hard to reverse with dietary changes or medication.

Another CRISPR-based technology is showing promise here. At the conference, Scribe said its in-house CasXE gene editor inactivates a gene that makes Lp(a) in liver cells. In mice, a single injection slashed levels of the fatty balls by up to 95 percent, with no detectable off-target editing.

Finally, the company showcased a different CasXE gene editor that kneecaps a gene associated with lipid production. Like other genetic targets, people with naturally lower levels of the gene APOC3 have low levels of blood lipids and lower risk of heart disease. One shot edited over 75 percent of all liver cells in monkeys and almost completely reversed high blood lipid levels in mice.

These are all preliminary results, but they could lead to a quantum shift in managing a global chronic disease with a single shot instead of daily pills.

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2025-11-26 00:17:34

From restoring movement and speech in people with paralysis to fighting depression, brain implants have fundamentally changed lives.

But inserting implants, however small or nimble, requires risky open-brain surgery. Pain, healing time, and potential infections aside, the risk limits the technology to only a handful of people.

Now, scientists at MIT Media Lab and collaborators hope to bring brain implants to the masses. They’ve created a tiny electronic chip powered by near-infrared light that can generate small electrical zaps. After linking with a type of immune cell to form bio-electronic hybrid chips, a single injection into the veins of mice shuttled the devices into their brains—no surgery required.

It sounds like science fiction, but the injected chips easily navigated the brain’s delicate and elaborate vessels to zero in on an inflamed site, where the microchip reliably delivered electrical pulses on demand. The chips happily cohabitated with surrounding neurons without changing the cells’ health or behavior.

“Our cell-electronics hybrid fuses the versatility of electronics with the biological transport and biochemical sensing prowess of living cells,” said study author Deblina Sarkar in a press release.

The strategy, which the researchers call circulatronics, could radically change brain stimulation. Targeted electrical zaps have shown early promise for treatment of a variety of brain diseases, such as Alzheimer’s, depression, and brain tumors.

And because the devices can be engineered to dissolve after a certain amount of time, they could potentially collect neural signals from healthy people, providing an unprecedented look into our brain’s inner workings.

A Long Road

Today’s brain implants are relatively bulky and struggle to reach deep into the brain. Most use batteries, either directly inside the device or in a battery pack affixed to the skull.

An ideal implant would be self-powered, controllable, and small enough to move through the smallest nooks and crannies of the brain and its vessels. A previous device, about the size of a grain of rice, used magnetic energy for power and generated electrical zaps in rodents while they actively roamed around. But because the device was controlled by magnetic fields, the setup required large and expensive hardware. Magnetic particles also tend to move in straight lines. This makes them terrible at navigating our brains serpentine vessels.

Near-infrared light offers an alternative to magnetic control. The wavelength easily penetrates the skull and brain with minimal scattering, suggesting it could control devices deep in the brain. Earlier this month, a team engineered an infrared-powered implant smaller than a grain of salt that could record from or stimulate neurons in mice. Although the device still required minimal surgery to implant, it reliably captured brain signals for a year, roughly half a mouse’s lifespan.

Infrared light has long been on Sarkar’s radar for an injectable brain implant. For six years, her team worked to solve multiple difficult roadblocks, eventually landing on circulatronics.

Tag Team

The team first had to make a chip so small it could easily flow through blood vessels without damaging them. The team turned to photovoltaic components that convert light into electricity, similar to the way solar panels work.

The chips are made of organic semiconductors that are biocompatible and flexible. This makes them suitable for navigation of our squishy bodies. Each one is like a tiny, light-powered battery sandwich, with a positive and negative metallic layer and an organic polymer inner filling.

Roughly 10 microns in diameter and smaller than a cell, these chips can be manufactured en masse with the same technology used to make computer chips. In tests with molds simulating the brain, the chips reliably generated electrical currents.

Then there was the problem of getting the chips to their target. The brain is protected by a wall of cells called the blood-brain barrier. The barrier is extremely selective of what molecules, proteins, and other materials can enter. Electronics, no matter how small, don’t make the cut. Some studies have tried to deliberately pry open the blood-brain barrier, but even a brief opening invites pathogens and other dangerous molecules inside.

The team’s solution was a cellular Trojan horse. When the brain experiences inflammation, the blood-brain barrier admits immune cells called monocytes. These cells roam the bloodstream equipped with chemical beacons to hunt down inflammatory sites. In theory, microchips could catch a ride on these cells through the blood-brain barrier without forcing it open.

To link monocytes to their tiny chip, the team used a Nobel Prize-winning technology called click chemistry. Think of it as Velcro. The researchers altered the surfaces of the monocytes in such a way that they formed Velcro-like “loops.” Then they added chemical “hooks” to the chips. When these components met, they clicked into place—but were still easily detachable—to form the final implant.

“The living cells camouflage the electronics so that they aren’t attacked by the body’s immune system, and they can travel seamlessly through the bloodstream. This also enables them to squeeze through the intact blood-brain barrier without the need to invasively open it,” said Sarkar.

Roaming Biohybrid Bots

To test their hybrid implants, the team tagged them with glow-in-the-dark trackers and injected them into the veins of mice. The critters had been given a chemical that triggered inflammation at a specific site deep in their brains.

Within 72 hours, the hybrid chips self-implanted into the inflamed area, whereas electronics lacking a cellular partner were barred from the brain. On average, around 14,000 hybrid implants latched onto the brain.

The devices worked as expected. After receiving pulses of near-infrared light for 20 minutes, neurons in the implanted region spiked with electrical activity at a magnitude similar to spikes trigged by current brain implants. Neighboring neurons were undisturbed.

The hybrid implants didn’t seem to affect the brain’s activity. Animals with the implant roamed around as usual. They showed no sign of changes to mood, memory, or other cognitive functions, happily sipping water and maintaining body weight for six months. Despite circulating in the blood after injection, the hybrid implants had no observable impact on other organs.

Although this study focused on brain inflammation, a similar strategy could be used to shuttle brain stimulation chips into stroke sites to aid rehabilitation. The system is relatively plug-and-play. Swapping monocytes for other cell types, such as T cells or neural stem cells, could allow them to act like cellular taxis for a wide range of other diseases.

The team hopes to kick off clinical trials of the technology within three years through MIT spinoff company, Cahira Technologies.

“This is a platform technology and may be employed to treat multiple brain diseases and mental illnesses,” said Sarkar. “Also, this technology is not just confined to the brain but could also be extended to other parts of the body in future.”

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