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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After a season of bingeing and drinking, your body may feel like it needs a break from the party.
Dry January — a modern trend that challenges people to abstain from drinking for the first month of the year — has become a popular way to “detox” from the holidays and start the new year on a healthy note.
Research has linked alcohol to a variety of health conditions, ranging from hangovers to higher cancer risk.
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In a recent podcast episode of “The Dr. Mark Hyman Show,” Dr. Mark Hyman, chief medical officer of Function Health, shared how 30 days of not drinking alcohol can transform health.
Hyman, who is based in Massachusetts, called Dry January a “powerful way to see in real time how alcohol affects nearly every system of your body and how quickly those systems can recover.”
Dry January has become a popular way to “detox” from the holidays and start the new year on a healthy note. (iStock)
Alcohol’s toll on the brain and body
Hyman acknowledged that most people drink to feel happier and more comfortable in social situations. This effect is caused by the main ingredient in alcohol, called ethanol, which can also have toxic effects.
Instead of stimulating the brain, alcohol slows it down and loosens inhibitions. “You feel more relaxed, more social, more confident, maybe you feel a little euphoric,” Hyman said.
Alcohol’s effect on the brain can also lead to poorer decisions and slower reflexes, the doctor cautioned.
Drinking alcohol can cause cognitive decline and brain fog, experts warn. (iStock)
Drinking also impacts the prefrontal cortex of the brain, which Hyman described as “the adult in the room,” responsible for judgment, planning and restraint. “It goes offline early in drinking, which explains why people feel freer or act impulsively when they drink,” he said.
Even moderate drinking can cause metabolic stress, inflammation, impaired detoxification and hormonal shifts, Hyman said, which can impact nearly every organ system in the body.
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Alcohol consumption has also been linked to an increased risk of cancer, metabolic dysfunction, gut microbiome disturbances and mitochondrial toxins.
It can also prevent the body from falling into REM sleep, which is the deep rest recovery period when the immune system cleans out the day’s toxins, according to Hyman.
Alcohol can impact deep rest and mental health, according to experts. (iStock)
Memory loss, cognitive decline, anxiety, sleep disruption, dementia and cardiovascular disease are all known risks of long-term alcohol use, as well as liver complications like fatty liver disease.
“Bottom line, alcohol taxes every major system in your body, especially your liver, your brain, your gut, your hormones,” Hyman said.
The effects of 30 days with no alcohol
The first week after your last drink, the body begins to detoxify and reset, according to Hyman. Blood sugar and cortisol stress hormones level out, and the liver begins to process a “backlog of toxins.” The body also re-hydrates and re-energizes.
The first 30 days with no alcohol allows the body to balance itself out. (iStock)
The second week, the gut and brain will begin to re-balance, as hormones like serotonin and dopamine stabilize, gut inflammation drops and the microbiome begins to heal. Cravings for sugar and alcohol will wane and mental clarity returns, the doctor said.
Week three is marked by further decreases in inflammation, fatty liver and blood pressure. This can be noticeable in the skin, as puffiness and redness are reduced. Mood also begins to stabilize, with lower anxiety levels.
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In week four, the body experiences additional metabolic and immune benefits, Hyman shared, including more insulin sensitivity, which makes it easier to lose weight.
“You have a stronger immune response. You’re not getting sick as much. You have better deep sleep, balanced hormones, especially cortisol and testosterone,” he said. “And you see a big change in energy, confidence and focus.”
Abstaining from alcohol can help restore energy, according to experts. (iStock)
Dr. Pinchieh Chiang, a clinician at Circle Medical in San Francisco, said that Dry January isn’t a “detox,” but rather provides “feedback” from the body.
“It gives the body time to show people how it feels without alcohol. For many, that insight alone changes their relationship with drinking,” she said. “The biggest surprise isn’t what people give up, it’s how much better they feel.”
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The doctor confirmed that the first few days of not drinking may feel harder than expected, sometimes causing restlessness, cravings or disrupted sleep, but Dry January can ultimately change drinking habits for the remainder of the year.
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After a full year without alcohol, Chiang noted that health improvements are more profound. “We see sustained improvements in blood pressure, liver function and inflammation,” she said. “Those changes directly affect long-term heart disease and stroke risk.”
The risks of ‘all or nothing’
Some experts warn that adopting the Dry January trend could strengthen the urge to drink more in the other months, noting that some drinkers may find more success by slowly consuming fewer drinks per week.
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Thomas Stopka, Ph.D., an epidemiologist and professor in the public health and community medicine department at Tufts University School of Medicine in Massachusetts, shared in a Futurity report that for some people, “damp January” may be more suitable.
One expert warned that not all drinkers should quit “cold turkey,” as it could lead to severe withdrawals. (iStock)
“Dry January is well-intentioned, and it may work really well for the people who can stick to it, maybe even beyond January,” he said. “Other people may be more inclined to cut down on alcohol consumption rather than quit drinking completely for the month.”
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Stopka noted that successful harm-reduction approaches “aim to be judgment free.”
“Substance use disorder is a disease,” he said. “It takes time to treat the disease and to stay connected to the continuum of care — from prevention to treatment initiation to sustained therapy, whether through medication, self-help, or individual therapy or group support.”
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Those struggling with signs of alcohol use disorder should consult a medical professional for personalized guidance.
Fox News Digital reached out to several alcohol industry associations requesting comment.
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