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

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

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4 minutes ago

February 27, 2026

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

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Health

‘SuperAgers’ stay mentally sharp well past 80, as scientists reveal the reason

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52 minutes ago

February 27, 2026

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‘SuperAgers’ stay mentally sharp well past 80, as scientists reveal the reason

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Stat of the week

More than 59% of women may have high blood pressure by 2050, according to a new report from the American Heart Association.

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This article was written by Fox News staff.

Health

Heart disease threat projected to climb sharply for key demographic

Published

13 hours ago

February 27, 2026

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Heart disease threat projected to climb sharply for key demographic

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A new report by the American Heart Association (AHA) included some troubling predictions for the future of women’s health.

The forecast, published in the journal Circulation on Wednesday, projected increases in various comorbidities in American females by 2050.

More than 59% of women were predicted to have high blood pressure, up from less than 49% currently.

The review also projected that more than 25% of women will have diabetes, compared to about 15% today, and more than 61% will have obesity, compared to 44% currently.

As a result of these risk factors, the prevalence of cardiovascular disease and stroke is expected to rise to 14.4% from 10.7%.

The prevalence of cardiovascular disease and stroke in women is expected to rise to 14.4% from 10.7% by 2050. (iStock)

Not all trends were negative, as unhealthy cholesterol prevalence is expected to drop to about 22% from more than 42% today, the report stated.

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Dr. Elizabeth Klodas, a cardiologist and founder of Step One Foods in Minnesota, commented on these “jarring findings.”

“The fact that on our current trajectory, cardiometabolic disease is projected to explode in women within one generation should be a huge wake-up call,” she told Fox News Digital.

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“Hypertension, diabetes, obesity — these are all major risk factors for heart disease, and we are already seeing what those risks are driving. Heart disease is the No. 1 killer of women, eclipsing all other causes of death, including breast cancer.”

Cardiovascular disease is the leading cause of death for women in the U.S. and around the world. (iStock)

Klodas warned that heart disease starts early, progresses “stealthily,” and can present “out of the blue in devastating ways.”

The AHA published another study on Thursday revealing one million hospitalizations, showing that heart attack deaths are climbing among adults below the age of 55.

The more alarming finding, according to Klodas, is that young women were found more likely to die after their first heart attack than men of the same age.

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“This is all especially tragic since heart disease is almost entirely preventable,” she said. “The earlier you start, the better.”

Children can show early evidence of plaque deposition in their arteries, which can be reversed through lifestyle changes if “undertaken early enough and aggressively enough,” according to the expert.

Moving more is one part of protecting a healthy heart, according to experts. (iStock)

Klodas suggested that rising heart conditions are associated with traditional risk factors, like smoking, high blood pressure, high cholesterol, diabetes, obesity and a sedentary lifestyle.

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Doctors are also seeing higher rates of preeclampsia, or high blood pressure during pregnancy, as well as gestational diabetes. Klodas noted that these are sex-specific risk factors that don’t typically contribute to complications until after menopause.

The best way to protect a healthy heart is to “do the basics,” Klodas recommended, including the following lifestyle habits.

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Klodas especially emphasized making improvements to diet, as the food people eat affects “every single risk factor that the AHA’s report highlights.”

“High blood pressure, high blood sugar, high cholesterol, excess weight – these are all conditions that are driven in part or in whole by food,” she said. “We eat multiple times every single day, which means what we eat has profound cumulative effects over time.”

“Even a small improvement in dietary intake, when maintained, can have a massive positive impact on health,” a doctor said. (iStock)

“Even a small improvement in dietary intake, when maintained, can have a massive positive impact on health.”

The doctor also recommends changing out a few snacks per day for healthier choices, which has been proven to “yield medication-level cholesterol reductions” in a month.

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“Keep up that small change and, over the course of a year, you could also lose 20 pounds and reduce your sodium intake enough to avoid blood pressure-lowering medications,” Klodas added.

“Women should not view the AHA report as inevitable. We have power over our health destinies. We just need to use it.”

Angelica Stabile is a lifestyle reporter for Fox News Digital.