Connect with us

Health

Mutated DNA Restored to Normal in Gene Therapy Advance

Published

on

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.

Advertisement

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.

Advertisement

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.

Advertisement

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.”

Advertisement

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.

Health

Controversial drug delivered rapid relief for severe depression in just hours

Published

on

Controversial drug delivered rapid relief for severe depression in just hours

NEWYou can now listen to Fox News articles!

Top stories

→ Single infusion of controversial drug changed severe depression symptoms within hours

→ What to know about thyroid cancer prognosis following Pam Bondi’s diagnosis

→ Tick bite ER visits are spiking as doctors warn of disease surge

Approximately 71 per 100,000 ER visits were tick-related in April, more than double the historical average. (iStock)

Advertisement

Health in the kitchen

→ Popular fruit may help protect your skin from the sun, new study suggests

→ Diet change tied to ‘younger’ biological age in older adults

→ Filtered water at specific ages could add months to lifespan decades later

CLICK HERE FOR MORE HEALTH STORIES

“The changes in gene expression [from eating grapes] indicated improvements in skin health,” one researcher said. (iStock)

Advertisement

Conversation starters

→ Finding sleep ‘sweet spot’ could help you live longer, study suggests

→ ‘Wild West’ peptide craze surges beyond GLP-1s, FDA to consider easing access

→ One type of sitting may pose greater dementia risk

Continue Reading

Health

Single infusion of controversial drug changed severe depression symptoms within hours, study finds

Published

on

Single infusion of controversial drug changed severe depression symptoms within hours, study finds

NEWYou can now listen to Fox News articles!

People experiencing severe depression with suicidal symptoms may not have to wait weeks for traditional antidepressants to take effect.

A recent review suggests that a single intravenous ketamine infusion can provide rapid relief for some patients.

Originally developed as an anesthetic, ketamine is a medicine that can reduce pain and, in some cases, help treat depression, but it can also be misused as a recreational drug, experts warn.

SINGLE DOSE OF POWERFUL PSYCHEDELIC CUTS DEPRESSION SYMPTOMS IN CLINICAL STUDY

Advertisement

Researchers from the University of Connecticut School of Medicine reviewed 26 clinical trials that included more than 1,100 patients. Approximately 626 received ketamine and 540 served as controls who did not take the drug.

Most of the trials included patients with major depressive disorder, but 11.5% included those with bipolar depression and 7.7% included people with both unipolar and bipolar depressive diagnoses.

A recent review suggests that a single intravenous ketamine infusion can provide rapid relief for some patients with treatment-resistant depression. (iStock)

Compared to a placebo, a single treatment significantly reduced depression in just four hours and dramatically lowered suicidal thoughts within 24 hours, the study found.

Patients reported fewer depressive symptoms after a week and reduced suicidal thoughts for up to a month after one ketamine infusion. Those who received repeated ketamine infusions showed a similar reduction of suicidal and depressive symptoms at the end of the treatment.

Advertisement

WHAT IS KETAMINE THERAPY? MORMON REALITY STARS TOUT CONTROVERSIAL TREATMENT

The most common adverse effects of ketamine – including headaches, numbness, dissociation (“out of body” experiences), nausea, dizziness and visual disturbances – were temporary and resolved within hours of the infusion.

Rarer, more serious side events included hospitalization, suicide attempts and suicide, but most were unrelated to ketamine, the review stated.

The analysis was published in May in JAMA Psychiatry.

Treatment-resistant depression

Major depressive disorder is a formal psychiatric diagnosis affecting approximately 280 million people globally, according to recent research.

Advertisement

Effective treatment involves a combination of therapy and medication, frequently antidepressants. However, for a few patients, symptoms do not respond to multiple therapies, a condition known as treatment-resistant depression, doctors say.

“When all existing treatment options fail, patients with severe depression could consider ketamine infusions.”

These patients are at a higher risk of very serious, sometimes tragic consequences, including suicidal thoughts, suicide attempts and death.

“When all existing treatment options fail, patients with severe depression could consider ketamine infusions,” lead author Taeho Greg Rhee, PhD, of the University of Connecticut School of Medicine, told Fox News Digital. “This is still a safer option when compared to electroconvulsive therapy (ECT).”

Compared to a placebo, a single treatment significantly reduced depression in just four hours and dramatically lowered suicidal thoughts within 24 hours, the study found. (iStock)

Advertisement

Traditional antidepressants stabilize mood by slowly elevating serotonin levels in the brain, but it can take weeks for the full effect to be achieved.

Ketamine, in contrast, works rapidly by blocking glutamate, a neurotransmitter that can impact emotions negatively when levels are too high in the brain, according to Cleveland Clinic.

Implications for care

The authors say their findings have two important potential clinical applications.

CLICK HERE FOR MORE HEALTH STORIES

First, ketamine’s rapid effects can be a life-saving treatment in the emergency room for patients presenting with suicidal ideation.

Advertisement

Experts caution that the medication should only be administered in closely monitored settings, such as clinics, to ensure safe treatment. (iStock)

Second, the effects of a single ketamine infusion are relatively short-lived – as almost all patients relapsed with depressive symptoms after a single infusion – so those with treatment-resistant depression will need repeated sessions.

“While intravenous ketamine is not yet FDA-approved for treating depression, it may still be used with off-label indications for those with severe depression and/or with a high risk of suicidal behaviors,” said Rhee.

Experts urge caution despite promise

Dr. Lama Bazzi, a psychiatrist in private practice in New York City, has had several patients receive ketamine infusions.

CLICK HERE TO SIGN UP FOR OUR HEALTH NEWSLETTER

Advertisement

“For a small subset of patients in a major depressive episode or struggling with suicidal thoughts, intravenous ketamine can be genuinely lifesaving,” Bazzi, who was not involved in the study, told Fox News Digital. “The relief they experience is almost immediate, offering them distance from the intensity of their emotions.”

However, she cautions that the medication should only be administered in closely monitored settings, such as clinics, to ensure safe treatment.

CLICK HERE TO DOWNLOAD THE FOX NEWS APP

Ketamine is not a panacea, Rhee agreed, warning of the potential risk of abuse and addiction.

“It should only be used medically,” he advised.

Advertisement

Ketamine’s rapid effects can be a life-saving treatment in the emergency room for patients presenting with suicidal ideation, some experts claim. (Getty Images)

Dr. Marc Siegel, Fox News senior medical analyst, noted in previous comments to Fox News Digital that ketamine is increasingly being used to treat severe depression, but emphasized that it should be administered under careful medical supervision because of its potential risks.

Study limitations

Although the studies compared ketamine with a placebo, some patients may have realized they were receiving the drug. This could have influenced how they reported their symptoms and how effective they perceived the treatment to be, according to the researchers.

“It should only be used medically.”

Another limitation is the small sample size of the studies, which could make the effects seem disproportionately magnified.

Advertisement

Also, as this was a review of many different studies, it is challenging to apply the findings to the general population, the researchers noted.

TEST YOURSELF WITH OUR LATEST LIFESTYLE QUIZ

“While long-term outcomes have not been studied, I believe that when patients are severely depressed or suicidal, ketamine is sometimes the only choice that almost always works,” Bazzi added.

Anyone interested in exploring alternative depression treatments should first consult a doctor.

Advertisement
Continue Reading

Health

Tick bite ER visits hit highest seasonal level in years as doctors warn of disease surge

Published

on

Tick bite ER visits hit highest seasonal level in years as doctors warn of disease surge

NEWYou can now listen to Fox News articles!

Tick bite-related ER visits are at their highest seasonal levels since 2017 across most U.S. regions, raising concerns about increased Lyme disease and other tick-borne illnesses.

That’s according to recent data from the Centers for Disease Control and Prevention’s Tick Bite Tracker, which monitors weekly emergency department visits associated with tick bites across the country.

For every 100,000 ER visits, approximately 71 were related to tick bites in April 2026, compared to a historical seasonal average of roughly 30 per 100,000.

DOCTORS REVEAL KEY SIGNS OF LYME DISEASE AS TICK SEASON INTENSIFIES ACROSS US

Advertisement

Some of the highest rates of tick-based ER visits were among children younger than 10 years and adults between 70 and 79 years.

A close-up shows a parasitic mite in motion on a human fingertip, highlighting the potential for disease transmission such as encephalitis. (iStock)

“Over the past three decades, the geographic range of the blacklegged tick has expanded significantly, and with it, the risk of Lyme disease and other Ixodes-transmitted infections,” Dr. Steven Goldberg, a family medicine physician who practices urgent care and family medicine at UofLHealth in Louisville, Kentucky, told Fox News Digital.

‘RABBIT FEVER’ CASES RISING IN US AS CDC WARNS OF ZOONOTIC BACTERIAL DISEASE

“The Ohio River Valley region is one of the most striking examples — Lyme disease cases in Ohio have increased roughly 10-fold over the past decade, likely driven by the convergence of Northeastern and Upper Midwestern tick populations meeting in that corridor.”

Advertisement

States like Virginia and West Virginia, as well as areas south of the traditional endemic zone, are reporting increasing tick abundance and disease cases, the doctor noted.

“Over the past three decades, the geographic range of the blacklegged tick has expanded significantly.”

“The lone star tick is also expanding its range northward beyond its traditional stronghold in the Southeast, which means diseases like ehrlichiosis and alpha-gal syndrome are appearing in regions where clinicians may not yet be thinking about them,” he warned.

Some climate studies predict that the blacklegged tick’s suitable habitat could expand by over 200% by the end of the century, Goldberg noted, including into Canada and across the central and southern U.S.

What’s driving the spike?

“Warmer, wetter conditions allow ticks to survive in habitats that previously would have been too cold,” said Dr. Suraj Saggar, chief of infectious disease at Holy Name Medical Center in Teaneck, New Jersey. “Milder winters also extend the lifespan of both ticks and the animals they feed on, accelerating tick reproduction and shortening their life cycles.”

Advertisement

Areas that historically experienced longer, colder winters or significant snow cover are now more hospitable to ticks, the doctor noted.

COPPERHEAD SNAKE BITE LEAVES MAYOR’S WIFE IN ‘EXCRUCIATING PAIN,’ HE REVEALS

“As temperatures rise and precipitation patterns change, ticks are able to spread northward and thrive in new ecosystems,” he said. 

Another contributing factor is increased land development and human expansion into wooded and grassy areas, as well as reforestation of formerly agricultural land.

“As temperatures rise and precipitation patterns change, ticks are able to spread northward and thrive in new ecosystems,” an expert said.  (iStock)

Advertisement

“The recovery and expansion of white-tailed deer populations — critical hosts for adult blacklegged ticks — has been a major driver,” Goldberg added. “Deer density is positively associated with Lyme disease incidence. Small mammal communities, particularly white-footed mice that serve as key reservoir hosts for Borrelia burgdorferi, also play a central role.”

Tick-borne diseases

Tick bites are known to transmit numerous illnesses, the most widespread of which is Lyme disease, a bacterial infection.

“Lyme disease cases alone have increased roughly two- to threefold over the past 20 years,” Saggar said. Approximately 476,000 Americans are diagnosed and treated for Lyme disease each year, per CDC surveillance data.

MOSQUITO-BORN DENGUE FEVER CASES SURGE AT POPULAR US VACATION DESTINATION

Also common are anaplasmosis and ehrlichiosis, two different types of bacterial infections, according to the doctor. Tick bites can also cause babesiosis, a malaria-like parasitic disease that infects and destroys red blood cells.

Advertisement

“Another growing concern is alpha-gal syndrome, a condition in which a (lone star) tick bite triggers a serious allergic reaction to red meat,” Saggar said. “In rare cases, people have died from anaphylactic reactions linked to alpha-gal syndrome following a tick bite.”

Some common symptoms of tick-borne illness include fever, chills, fatigue, headaches, muscle aches and joint pain. (iStock)

Ticks can also transmit viruses, including the Powassan virus, which can cause severe neurologic injury.

“Powassan virus disease is arguably the most concerning emerging tick-borne infection,” said Goldberg, who is also chief medical officer at HealthTrack. “It’s transmitted by the same blacklegged tick that carries Lyme disease, but unlike Lyme, it can be transmitted within minutes of tick attachment.”

CLICK HERE TO DOWNLOAD THE FOX NEWS APP

Advertisement

Powassan can cause severe encephalitis with a roughly 10% to 15% fatality rate, and more than half of survivors have lasting neurological deficits, Goldberg noted.

In the Rocky Mountain states, the Rocky Mountain wood tick (Dermacentor andersoni) transmits Rocky Mountain spotted fever and Colorado tick fever.

CLICK HERE FOR MORE HEALTH STORIES

“In the Southeast and South-Central U.S., the lone star tick (Amblyomma americanum) drives a different set of concerns: ehrlichiosis, tularemia, and two emerging viral threats — Heartland virus and Bourbon virus,” said Goldberg.

Symptoms to watch for

Some common symptoms of tick-borne illness include fever, chills, fatigue, headaches, muscle aches and joint pain, according to Saggar.

Advertisement

Another sign is the classic “bull’s-eye” rash associated with Lyme disease, known medically as “erythema migrans.” 

“If you think you have been bitten by a tick, you should seek medical attention if you develop symptoms after a known tick bite or after spending time in tick-prone areas, especially during the spring, summer and fall.” (iStock)

“Because testing can sometimes be falsely negative early in the disease process, doctors may treat patients based on symptoms and exposure history rather than waiting for laboratory confirmation,” Saggar noted. 

TEST YOURSELF WITH OUR LATEST LIFESTYLE QUIZ

“If you think you have been bitten by a tick, you should seek medical attention if you develop symptoms after a known tick bite or after spending time in tick-prone areas, especially during the spring, summer and fall.”

Advertisement

Preventing tick bites

As there are no vaccines currently available for any tick-borne disease in the U.S., prevention is the most effective strategy.

Goldberg shared the following recommended prevention strategies.

CLICK HERE TO SIGN UP FOR OUR HEALTH NEWSLETTER

  • Use EPA-approved repellents, including DEET, picaridin, IR3535 or oil of lemon eucalyptus on exposed skin. Treat clothing and gear with permethrin (a synthetic insecticide and repellent) or purchase pre-treated clothing.
  • Wear light-colored clothing (to spot ticks more easily), long sleeves and long pants tucked into socks when in wooded or grassy areas.
  • After spending time outdoors, check your entire body, paying special attention to the scalp, behind the ears, armpits, groin and behind the knees, the doctor advised. It’s also recommended to shower within two hours of coming indoors.
  • Tumble-dry clothing on high heat for at least 10 minutes to kill any ticks on clothing.
  • Remove ticks promptly and properly. Using fine-tipped tweezers, grasp the tick as close to the skin as possible, and pull upward with steady, even pressure. Clean the bite area afterward.

Approximately 476,000 Americans are diagnosed and treated for Lyme disease each year, per CDC data.

“The longer a tick is attached, the higher the risk of disease transmission — for Lyme disease, transmission generally requires at least 36 hours of attachment,” Goldberg said. “The Powassan virus can be transmitted much more quickly.”

Advertisement

Continue Reading
Advertisement

Trending