Health

Mutated DNA Restored to Normal in Gene Therapy Advance

Published

9 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

5 winter-weather essentials to protect skin health in dangerously cold temperatures

Published

9 hours ago

December 14, 2025

Press Room

5 winter-weather essentials to protect skin health in dangerously cold temperatures

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As winter weather moves in, cozy essentials like scarves, plush throws and heated bedding become everyday comforts.

But dermatologists caution that these cold-weather favorites can secretly undermine skin health — trapping sweat and bacteria, causing irritation and exposing the skin to excess heat.

Choosing the right materials for wellness — and using them safely — can make a big difference.

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Here are five winter must-haves and gift ideas, with expert tips on how to keep skin healthy and happy while staying warm this season.

Soft scarf, $19.99, Amazon.com

Scarves made of natural fiber, like this one that is 100% cotton, may help if you’re prone to irritation. (Amazon)

A soft scarf is a staple for cold days and an easy way to elevate a winter outfit.

Yet, if breakouts are appearing along the neck, jawline or chest, that favorite accessory may be part of the problem.

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“You should wash your scarf as often as your clothes to prevent breakouts,” Annabelle Taurua, a beauty expert at Fresha, a beauty and wellness booking platform headquartered in London, told Fox News Digital.

Cotton is a better choice than polyester, she also noted, as it’s more breathable and allows sweat to evaporate.

Fluffy blankets, $28.97, Potterybarn.com

Fluffy blankets make for perfect cozy days at home, but their soft fibers can trap sweat, oils and dead skin — which can clog pores and encourage bacteria growth. (Pottery Barn)

Fluffy blankets make for perfect cozy days at home, but their soft fibers can trap sweat, oils and dead skin, which can clog pores and encourage bacteria growth.

Rough textures or infrequent washing can also irritate sensitive skin and worsen breakouts.

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“Regularly wash your blankets, especially those made from synthetic or fluffy materials, to remove built-up oils and dirt,” Taurua advised.

She recommended breathable materials like cotton or linen, as well as hypoallergenic options for anyone prone to irritation.

Good set of sheets, $49.99, Amazon.com

Much like scarves, natural fibers are the way to go for your linens to avoid irritation. (iStock)

Cold weather makes lingering in bed especially tempting, but lying on unwashed bedding can worsen acne.

Pillowcases and sheets quickly collect oil, bacteria and dead skin cells, which transfer directly onto the face.

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“Washing bedding frequently is important,” Taurua said. “You should aim to change and wash your pillowcases every two to three days and your sheets at least once a week.”

Heated blanket, $33.99, Walmart.com

Heated blankets are a great way to stay warm during the winter. (Walmart)

When using a heated blanket, start with the lowest heat setting and limit use, said Taurua.

“Once you’re warm, switch to a regular blanket,” she advised.

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She also said it’s best to avoid direct contact for long periods to reduce irritation.

Hot water bottle, $12.99, Amazon.com

A traditional winter staple, hot water bottles offer quick comfort — but they come with risks similar to heated blankets, including burns, scalding and long-term heat-related skin damage.

Hot water bottles should never be filled with boiling water, experts advise. (Amazon)

“Never fill a hot water bottle with boiling water,” Taurua said.

“Only use hot, not boiling, water, and fill it to a maximum of two-thirds.”

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She recommended wrapping the bottle in a towel or cover to avoid direct skin contact and limiting use to around 20 minutes.

Khloe Quill is a lifestyle production assistant with Fox News Digital. She and the lifestyle team cover a range of story topics including food and drink, travel, and health.

Health

Foods and Drinks That Help With Bloating: Flatten Your Belly in Days

Published

20 hours ago

December 13, 2025

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Foods and Drinks That Help With Bloating: Flatten Your Belly in Days

Foods That Help With Bloating and Shrink Your Waist Fast | Woman’s World

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Health

What to know about Cushing’s syndrome, which led to Amy Schumer’s dramatic weight loss

Published

21 hours ago

December 13, 2025

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What to know about Cushing’s syndrome, which led to Amy Schumer’s dramatic weight loss

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Comedian Amy Schumer, 44, has recently come clean about her struggle with Cushing’s syndrome, leading to a dramatic weight loss.

Schumer’s transformation has sparked conversation online, to which she responded in a now-deleted Instagram post that shut down speculation about cosmetic enhancements.

“I didn’t lose 30lbs — I lost 50,” she emphasized on social media, adding that she does not get Botox or filler.

AMY SCHUMER DROPPED 50 POUNDS TO BATTLE DISEASE THAT ‘CAN KILL YOU’ IF UNTREATED

Schumer doubled down that her weight loss was not about her appearance, but about staying alive.

“Not to look hot, which does feel fun and temporary,” she said. “I did it to survive. I had a disease that makes your face extremely puffy that can kill you, but the internet caught it and that disease has cleared.”

Amy Schumer attends Variety’s 2024 Power of Women: New York event on May 2, 2024, in New York City on the left. On the right, Schumer later poses for a photo posted to Instagram after her weight loss. (Marleen Moise/WireImage; Amy Schumer/Instagram)

“Sorry for whatever feeling it’s giving you that I lost that weight,” she added. “I’ve had plastic surgery over the years and I use [Mounjaro]. Sorry to anyone they let down. I’m pain free. I can [play] tag with my son.”

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The actress also addressed her shifting hormones, saying that she’s “happy to share more if anyone has any questions about how I’m looking or feeling or where I am in my perimenopause process.”

“I didn’t lose 30lbs — I lost 50,” Schumer emphasized on social media, adding that she does not get Botox or filler. (Amy Schumer/Instagram)

What is Cushing’s syndrome?

Schumer previously revealed that she had been diagnosed with Cushing’s syndrome, a hormonal disorder that can cause extreme swelling, fatigue and potentially fatal complications.

Dr. Peter Balazs, a hormone and weight loss specialist in New Jersey, provided more details on the condition in an interview with Fox News Digital.

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“Cushing’s syndrome is caused by prolonged, high exposure to cortisol, which is your body’s main stress hormone,” he said. “Usually, the excess cortisol is a result of prolonged steroid use. We see this many times with patients who have some autoimmune disorder.”

Balazs said the “key” to Cushing’s syndrome is not just weight gain, but a “specific redistribution” of fat caused by too much cortisol.

Amy Schumer is photographed during a guest interview on The Tonight Show Starring Jimmy Fallon on Feb. 13, 2024 (left), and later seen walking through midtown New York City on Oct. 28, 2025 (right). (Todd Owyoung/NBC via Getty Images; Raymond Hall/GC Images/Getty Images)

Fat may be driven to the abdomen, chest, upper back (referred to as a “buffalo hump”) or face, sometimes considered “moon face,” the doctor said.

Cortisol also breaks down protein, which leads to a thinning in the arms and legs. “Weight gain can be tough, involuntary and hard to manage,” Balazs added.

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Cortisol stimulates “hunger centers” in the brain, raises blood sugar and promotes fat storage, especially deep belly fat, according to the doctor.

Cushing’s syndrome can also cause high blood pressure, bone loss and sometimes type 2 diabetes, per Mayo Clinic.

In addition to weight gain in certain areas, other symptoms may include pink or purple stretch marks on the stomach, hips, thighs, breasts and underarms, as well as acne, slow wound healing, and thin, frail skin that bruises easily.

With Cushing’s syndrome, fat may accumulate in the abdomen, chest, face or upper back (referred to as a “buffalo hump”). (iStock)

Women with Cushing’s syndrome often experience thick, dark hair on the face and body, as well as irregular periods. Symptoms among men can include a lower sex drive, reduced fertility and erectile dysfunction.

Mayo Clinic listed other potential symptoms, including extreme fatigue, muscle weakness, depression, anxiety, irritability, mood swings, trouble with concentration and memory, headache, sleeplessness, skin darkening and stunted growth in children.

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Those with symptoms should contact their doctor immediately, especially if they are taking steroids to treat health issues like asthma, arthritis or inflammatory bowel disease, as these medications can exacerbate the condition.

The Mayo Clinic has reported that the “sooner treatment starts, the better the chances for recovery.”

“I’ve had plastic surgery over the years and I use [Mounjaro],” Schumer stated in a social media post. (George Frey/Bloomberg)

For patients like Schumer, weight loss is typically not the main goal, Balazs noted, but it is a “critical sign” of successful treatment.

“The primary goal is to normalize your high cortisol levels,” he said. “I believe Amy Schumer got treated first for her underlying problem. Once the cortisol is normalized, which is the most important step, there’s a role to use adjunct medications to decrease weight.”

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Among GLP-1s, Mounjaro is an “excellent choice to decrease deep belly fat and increase insulin sensitivity of cells,” Balazs added.

Fox News Digital’s Stephanie Giang-Paunon contributed to this report.

Fox News Digital reached out to Schumer’s rep requesting comment.

Angelica Stabile is a lifestyle reporter for Fox News Digital.