Health
Mutated DNA Restored to Normal in Gene Therapy Advance
Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene.
This was the first time a mutated gene has been restored to normal.
The small study of nine patients announced Monday by the company Beam Therapeutics of Cambridge, Mass., involved fixing a spelling error involving the four base sequences — G, A, C and T — in DNA. The effect was to change an incorrect DNA letter to the right one. The result was a normal gene that functioned as it should, potentially halting liver and lung damage of patients with a rare disorder.
“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine.
He added that the method offers the hope of treating other genetic diseases precisely by fixing mutations — an alternative to current gene therapies, which either add new genes to compensate for mutated ones, or slicing DNA to silence genes.
Dr. Musunuru is a co-founder and equity holder of Verve Therapeutics, a gene therapy company, and receives funding from Beam Therapeutics for research, but not for this study.
Dr. Richard P. Lifton, president of Rockefeller University and head of its Laboratory of Human Genetics and Genomics, said the sort of gene editing Beam did, rewriting genes with an infusion, “is a holy grail” that “has the promise for being a one-and-done kind of therapy.”
Dr. Lifton is a director of Roche Pharmaceuticals and its subsidiary Genentech.
Despite the study’s small size, he said the results are “a very impressive advance and very promising.”
The study involved patients who have alpha-1 antitrypsin deficiency, or AATD, a genetic disease that affects an estimated 100,000 Americans, mostly of European ancestry. That makes it as common as sickle cell in this country. It is progressive and incurable.
The alpha-1 antitrypsin protein is made in the liver and normally goes to the lungs and protects them from inflammation from inhaled irritants like smoke or dust. But in people with the disease, a single change in a DNA letter in the gene results in a misshapen and nonfunctional protein. The result is often emphysema or chronic obstructive pulmonary disease in unprotected lungs.
But many of the aberrant alpha-1 antitrypsin proteins never get to the lungs and instead build up in patients’ livers, often causing cirrhosis.
The gene editing was simple for patients. They sat in a chair for a couple of hours while lipid nanoparticles, like those used in Covid vaccines, were infused into their blood. The nanoparticles did not hold vaccines, though. Instead, they encased a microscopic gene editor. The lipid casing protected the editor on a journey to the liver.
When the nanoparticles reached the liver, the lipid layer peeled off, releasing the editor — a disabled CRISPR molecule that acted like a GPS for the genome and an enzyme to fix the mutation. The CRISPR molecule crawled along the patient’s DNA until it found the one incorrect letter that needed to be repaired among the three billion DNA letters in the genome. Then the editing enzyme replaced that letter with the correct one.
The study divided the patients into three groups and tested three different doses of the gene editor. Those who got the highest dose made enough normal alpha-1 antitrypsin to be in a range where no more damage should occur. There were no serious side effects, said John Evans, Beam’s chief executive officer.
Beam will now be offering the higher dose to the patients who got the lower doses in the company’s study. Beam will also study the treatment in more patients, and test an even higher dose of its gene editor.
“And then we immediately have to think about how we can get this approved,” Mr. Evans said.
Dr. Noel McElvaney, a professor at the Royal College of Surgeons in Ireland and an investigator in the Beam study, said there’s a real need for an effective treatment to halt the damage done by the mutated gene. He said he sees patients in their 30s and 40s with severe emphysema and “really bad liver disease.” And, he said, “by the time we see them there is already a significant amount of damage.”
For those suffering the worst effects of AATD, he said, the new gene therapy is “a major major breakthrough.”
“The big pro” of the new treatment, he said, is that “it theoretically cures the liver and lung disease in one go.”
Dr. McElvaney added, though, that “like all genetic interventions, we have to follow up for a long time to make sure it’s as good as we think it is.”
But patients now have renewed hope, said Dr. Andrew Wilson, scientific director of the Alpha-1 Foundation, an advocacy group.
“We have been dreaming of gene therapy as a treatment for this disease,” he said.
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The key to feeling better in a fast, overstimulated world might be surprisingly simple: Live a little more like your grandparents.
A growing social media trend, dubbed “nonnamaxxing,” draws inspiration from the slower, more intentional rhythms associated with an Italian grandmother.
The lifestyle is often linked to activities like preparing home-cooked meals, spending time outdoors and making meaningful connections.
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“Nonnamaxxing is a 2026 trend that embraces the slower, more intentional lifestyle of an Italian grandmother (a Nonna). Think cooking from scratch, long family meals, daily walks, gardening and less screen time,” Erin Palinski-Wade, a New Jersey-based registered dietitian, told Fox News Digital.
Nonnamaxxing, derived from the name for an Italian grandmother, is a trend that incorporates lifestyle habits hundreds of years in the making. (iStock)
Stepping away from screens and toward real-world interaction can have measurable benefits, according to California-based psychotherapist Laurie Singer.
“We know that interacting with others in person, rather than spending time on screens, significantly improves mental health,” she told Fox News Digital, adding that social media often fuels comparison and lowers self-esteem.
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Living more like previous generations isn’t purely driven by nostalgia. Cooking meals from scratch, for example, has been linked to better nutrition and more mindful eating patterns.
Adopting traditional mealtime habits can improve diet quality and support both physical and mental health, especially when meals are shared regularly with others, Palinski-Wade noted.
One longevity expert stresses that staying healthy isn’t just about food — it’s also about joy and community. (iStock)
There’s also a psychological benefit to slowing down and focusing on one task at a time. Anxiety often stems from unfinished or avoided tasks, Singer noted, and engaging in hands-on activities can counteract that.
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“Nonnamaxxing encourages us to be present around a task, like gardening, baking or knitting, or just taking a mindful walk, that delivers something ‘real,’” she said.
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Palinski-Wade cautions against turning the trend into another source of pressure, noting that a traditional “nonna” lifestyle often assumes a different pace of life.
The key, she said, is adapting the mindset, not replicating it perfectly.
Nonnamaxxing, derived from the name for an Italian grandmother, is a trend that incorporates lifestyle habits hundreds of years in the making. (iStock)
The goal is to reintroduce small, intentional moments that make you feel better.
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That might mean prioritizing a few shared meals each week, taking a walk without your phone or setting aside time for a simple hobby, the expert recommended.
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Singer added, “Having a positive place to escape to, through whatever activities speak to us and make us happy, isn’t generational – it’s human.”
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Feeling lonely may take a toll on older adults’ memory — but it may not speed up cognitive decline, according to a new study.
Researchers from Colombia, Spain and Sweden analyzed data from more than 10,000 adults ages 65 to 94 across 12 European countries and found those who reported higher levels of loneliness did worse on memory tests at the start of the study, according to research published this month in the journal Aging & Mental Health.
Over a seven-year period, however, memory decline occurred at a similar rate regardless of how lonely participants felt.
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“The finding that loneliness significantly impacted memory, but not the speed of decline in memory over time was a surprising outcome,” lead author Dr. Luis Carlos Venegas-Sanabria of the School of Medicine and Health Sciences at the Universidad del Rosario said in a statement.
Loneliness may be linked to memory performance in older adults, a new study suggests. (iStock)
“It suggests that loneliness may play a more prominent role in the initial state of memory than in its progressive decline,” Venegas-Sanabria said, adding that the findings highlight the importance of addressing loneliness as a factor in cognitive performance.
The findings add to debate about whether loneliness contributes to dementia risk. While loneliness and social isolation are often considered risk factors for cognitive decline, research results have been mixed.
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The study looked at data from the long-running Survey of Health, Ageing and Retirement in Europe (SHARE), which tracked 10,217 older adults between 2012 and 2019. Participants were asked to recall words immediately and after a delay to measure memory performance.
Social isolation and loneliness could play a surprising role in cognitive health among seniors. (iStock)
Loneliness was assessed using three questions about how often participants felt isolated, left out or lacking companionship.
About 8% of participants reported high levels of loneliness at the outset. That group tended to be older, more likely to be female and more likely to have conditions such as depression.
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Researchers found that those with higher loneliness had lower scores on both immediate and delayed memory tests at baseline. Still, all groups — regardless of loneliness level — experienced similar declines in memory over time.
The results suggest loneliness may not directly accelerate the progression of memory loss, though it remains linked to poorer cognitive performance overall.
Researchers look at a brain scan at the National Institutes of Health in Bethesda, Maryland. (Saul Loeb/AFP/Getty Images)
Experts warn, however, that the findings should not be interpreted to mean loneliness is harmless.
“The finding that lonely older adults start with worse memory but don’t decline faster is actually the most interesting part of the paper, and I think it’s easy to misread,” said Jordan Weiss, Ph.D., a scientific advisor and aging expert at Assisted Living Magazine and a professor at NYU Grossman School of Medicine.
“It likely means loneliness does its damage earlier in life, well before people show up in a study like this at 65-plus,” Weiss told Fox News Digital.
By older age, long-term social patterns may already be established, making it harder to detect when the effects of loneliness first took hold, an aging expert says. (iStock)
He suggested that by older age, long-term social patterns may already be established, making it harder to detect when the effects of loneliness first took hold.
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“By the time you’re measuring someone in their late 60s, decades of social connection patterns are already baked in,” he said.
Weiss, who was not involved in the research, added that loneliness may coincide with other health conditions, and noted that participants who felt more isolated also had higher rates of depression, high-blood pressure and diabetes. The link, he said, may reflect a cluster of health risks rather than a direct cause.
“While they can go hand-in-hand, it’s not clear that loneliness contributes to dementia,” a psychotherapist says. (iStock)
Amy Morin, a Florida-based psychotherapist and author, said the findings reflect a broader pattern in research on loneliness and brain health, and that the relationship may be more complex than it appears.
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“The evidence shows there’s a link between loneliness and cognitive decline but there’s no direct evidence of a cause and effect relationship,” she said. “So while they can go hand-in-hand, it’s not clear that loneliness contributes to dementia.”
Morin added that loneliness, which can fluctuate, may not be the root of the problem, but rather a symptom of other underlying mental or physical health issues.
Researchers suggested screening for loneliness be incorporated into routine cognitive assessments as one way to support healthy aging. (iStock)
She said staying socially and mentally engaged is crucial for overall brain health.
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“It’s important to be proactive about social activities,” Morin said. “Joining a book club, having coffee with a friend, or attending faith-based services can be a powerful way to maintain connections in older age.”
The researchers also suggested screening for loneliness be incorporated into routine cognitive assessments as one way to support healthy aging.
Fox News Digital reached out to the researchers for comment.
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