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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Wii Bowling Takes Over Tulsa Retirement Homes
Retirement communities in Tulsa, Okla., compete against one another in a Nintendo Wii Sports bowling league.
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“That’s how you win.” “There you go, Ron.” “Way to go.”
By Nick Oxford, Alisa Shodiyev Kaff and Alexandra E. Petri
June 19, 2026
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As the popularity of GLP-1 drugs continues to climb, significant weight loss associated with the medications has been linked to a growing list of cosmetic concerns.
Some surgeons report that more patients are seeking treatments for so-called “Ozempic earlobes,” which reportedly appear thinner, longer or more sagging after the loss of facial fat.
“The use of semaglutides causes you to lose fat across your body, including the small, fat pads of the earlobes,” facial plastic surgeon Sachin S. Parikh, MD, told NewBeauty.
OZEMPIC, OTHER SEMAGLUTIDES LINKED TO HAIR LOSS: HERE’S WHAT TO KNOW
“It’s important to note that semaglutides do not damage ear tissue or affect hearing in any way, so any intervention would be purely cosmetic,” added the California-based doctor.
Some of the potential treatments for “Ozempic earlobes” may include dermal filler, fat transfer, laser treatments or surgical earlobe reduction, according to the report.
As the popularity of GLP-1 drugs continues to climb, significant weight loss associated with the medications has been linked to a growing list of cosmetic concerns. (iStock)
Dr. Mohammed Asif from Duly Health and Care in Naperville, Illinois, said that while he hasn’t personally noticed an uptick in ear surgeries, he has seen a rise in other procedures due to GLP-1-triggered weight loss.
Some of those include panniculectomies (Ozempic skin removal surgery) and abdominoplasties (tummy tucks).
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“There has been a significant increase in body contouring procedures among patients with GLP-1 weight loss,” Asif told Fox News Digital.
“In my practice, I’ve seen a significant surge in breast lifts, panniculectomies (skin removal surgery), abdominoplasties (tummy tucks), brachioplasties (arm lifts) and thighplasties due to weight loss.”
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Healing and recovery are “far greater and less complicated” than with bariatric surgery weight loss, Asif noted.
“This is due to the gradual weight loss achieved over time with GLP-1s.”
“Ozempic breast” is another common complaint, according to Dr. Michael Omidi, a double board-certified plastic surgeon practicing in Beverly Hills.
“There has been a significant increase in body contouring procedures among patients with GLP-1 weight loss,” a doctor told Fox News Digital. (iStock)
“Women in their 30s and 40s would not typically need a breast lift, but when you’re taking drugs like Ozempic or Wegovy that cause rapid weight loss, women can see gravity take its toll on their breasts,” he told Fox News Digital.
“When women, especially younger ones, lose fat too fast, it causes the breasts, which are comprised of fat, glandular tissue and skin, to lose volume,” he went on. “The skin and supporting ligaments don’t always bouce back at the same rate as the weight loss, resulting in breasts that can appear saggy and deflated.”
NEW OBESITY TREATMENT MAY HELP PRESERVE MUSCLE DURING WEIGHT LOSS
Dr. Samuel Golpanian, a double board-certified plastic surgeon in Beverly Hills, said he has seen “dozens” of patients – men and women alike – seeking treatments for so-called “Ozempic butt” after significant weight loss.
“This is an unintended consequence of taking GLP-1s,” he told Fox News Digital.
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“Such dramatic weight loss in a short period of time causes the skin not to tighten quickly enough to keep up with the body’s changes. Our buttocks have a large amount of fat, so when that volume disappears quickly, you’ll develop a flatter, saggier behind.”
“Such dramatic weight loss in a short period of time causes the skin not to tighten quickly enough to keep up with the body’s changes,” a surgeon said. (iStock)
Kristy Hamilton, MD, a Houston-based surgeon who is a member of the American Society of Plastic Surgeons (ASPS), agreed that a growing number of patients are seeking skin-tightening procedures after losing large amounts of weight with GLP-1s.
“We’re absolutely seeing more of those patients, and I expect that trend to continue,” said Hamilton in an ASPS report.
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“People are losing as much as 100 pounds on this medication – and when you’re losing that amount, that’s significant. You’re certainly going to have excess skin afterward.”
The ASPS lists the following body-contouring procedures as the ones most frequently performed after substantial weight loss.
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- Tummy tuck (abdominoplasty): Removes excess abdominal skin and fat
- Lower body lift (belt lipectomy): Addresses the abdomen, buttocks, hips and thighs
- Arm lift (brachioplasty): Removes hanging upper-arm skin
- Thigh lift: Tightens excess skin of the inner thighs
- Breast lift (mastopexy): Addresses sagging, deflated breasts after weight loss
- Facelift/neck lift: Addresses facial volume loss and skin laxity after major weight loss
“People are losing as much as 100 pounds on this medication – and when you’re losing that amount, that’s significant. You’re certainly going to have excess skin afterward.” (iStock)
Experts say adequate protein intake and resistance training can help minimize the loss of lean muscle mass that often accompanies rapid weight loss, which could reduce some of the cosmetic concerns.
Golpanian also emphasized the importance of eating enough protein.
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“It can be harder since your appetite is suppressed, but it’s so important to consume at least 110 grams of protein a day while taking a GLP-1 drug,” he advised.
“Also, I say this to my patients all the time: You have to lift weights to keep your muscles from atrophying. Strength training can help build muscles and preserve your glutes.”
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