Health

Mutated DNA Restored to Normal in Gene Therapy Advance

Published

12 months ago

March 11, 2025

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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Health

Common nighttime noise exposure may trigger heart problems, study suggests

Published

7 hours ago

February 28, 2026

Press Room

Common nighttime noise exposure may trigger heart problems, study suggests

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Living near heavy traffic could negatively impact your heart health.

A European study, published in the journal Environmental Research, found that exposure to nighttime road traffic noise is linked to changes in the blood, leading to worsened cholesterol and cardiovascular risks.

The researchers considered data from the U.K. Biobank, Rotterdam Study, and Northern Finland Birth Cohort 1966, including more than 272,000 adults over the age of 30, according to a press release.

Nighttime road noise exposure was estimated at all participants’ homes based on national noise maps. Researchers also took blood samples to measure the participants’ metabolic biomarkers for disease, then mapped the link between nightly noise levels and existence of biomarkers.

Exposure to loud noise was associated with increased concentrations of cholesterol-related biomarkers. (iStock)

The study found that people exposed to louder noise at night — especially sounds above 55 decibels — showed changes in 48 different substances in their blood. Twenty of these associations “remained robust” throughout all cohorts.

Exposure to loud noise was associated with increased concentrations of cholesterol-related biomarkers, especially LDL “bad” cholesterol, IDL (intermediate-density lipoprotein) and unsaturated fatty acids.

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As noise levels increased, starting at around 50 decibels, cholesterol markers rose steadily, the release stated.

The authors concluded that this study “provides evidence that nighttime road traffic noise exposure from 50 dB upward is associated with alterations in blood cholesterol and lipid profiles in adults.”

Researchers noted a link between traffic noise and cardiometabolic disease. (iStock)

Study co-author Yiyan He, doctoral researcher at the University of Oulu in Finland, noted that in this type of research, small effect sizes are expected, and environmental exposures such as traffic noise are “typically modest.”

SIMPLE NIGHTLY HABIT LINKED TO HEALTHIER BLOOD PRESSURE, STUDY SUGGESTS

“Despite this, we observed statistically robust and consistent associations across many biomarkers, especially those related to LDL and IDL lipoproteins,” she told Fox News Digital.

MISSING SLEEP MAY TAKE A HIDDEN TOLL ON YOUR BRAIN AND LONGEVITY, RESEARCH REVEALS

“We also identified a clear exposure-response pattern starting at around 50 dB, suggesting that metabolic changes become more evident as noise levels increase.”

This aligns with public health guidance, as the World Health Organization recommends lower nighttime noise limits at around 40 to 45 dB, Yiyan He added.

“This finding may clarify the association between traffic noise and cardiometabolic diseases,” the researchers wrote. (iStock)

“The 55 dB level is often used as an interim benchmark associated with substantial noise annoyance and sleep disturbance,” she said. “In our study, we observed associations not only at 55 dB, but also indications of effects emerging at around 50 dB.”

The strength and consistency of the cholesterol-related associations were surprising, as these changes are usually “subtle.”

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“Instead, we found consistent associations across multiple large European cohorts, which strengthens confidence that the findings may reflect real biological patterns,” Yiyan He went on. “We were also interested to see that effects were minimal below ~50 dB, suggesting a possible threshold-like pattern.”

HEART DISEASE THREAT PROJECTED TO CLIMB SHARPLY FOR KEY DEMOGRAPHIC

The researcher noted that these findings were consistent across genders, education levels and obesity status.

The study was restricted to White Europeans, which posed a limitation. There was also a lack of information on the fasting status in the UK Biobank.

Changes in cholesterol levels were more severe than researchers expected. (iStock)

“Fasting can influence levels of certain metabolites, particularly fatty acids,” Yiyan He said. “However, based on UK Biobank documentation, fewer than 10% of participants were fasting for at least eight hours, and our main findings focused on cholesterol-related biomarkers, which are generally less sensitive to short-term fasting.”

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The researchers also lacked information on bedroom location, indoor noise exposure and time spent at home.

“These factors may introduce non-differential exposure misclassification,” Yiyan He said. “Additionally, noise exposure estimates were based on participants’ temporary residential addresses at the time of blood sampling, without considering the duration of residence.”

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“Many of these limitations would tend to bias results toward the null, so the consistent associations we observed remain noteworthy.”

Experts recommend taking measures to limit traffic noise at night. (iStock)

Based on this latest research, Yiyan He noted that nighttime noise is a “health-relevant exposure,” not just “an annoyance.”

“Our findings suggest that nighttime traffic noise may subtly but consistently affect metabolic health,” she said. “While the changes in cholesterol and lipid levels for any one individual are small, traffic noise affects a very large number of people, which means the potential public health impact could be substantial.”

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The researcher recommends taking measures like improving sound insulation, using noise-reducing strategies and placing bedrooms on the quieter side of the home when possible.

“Because sleep is a key pathway linking noise to health, protecting the nighttime sleep environment is especially important,” she added.

Angelica Stabile is a lifestyle reporter for Fox News Digital.

Health

The Best Time To Drink Coffee for Weight Loss and a Faster Metabolism

Published

18 hours ago

February 27, 2026

Press Room

The Best Time To Drink Coffee for Weight Loss and a Faster Metabolism

The Best Time To Drink Coffee for Weight Loss and Fat Burn | Woman’s World

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