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.
Use left and right arrow keys to navigate between menu items.
Use escape to exit the menu.
Sign Up
Create a free account to access exclusive content, play games, solve puzzles, test your pop-culture knowledge and receive special offers.
Already have an account? Login
Health
Parasitic infection causing ‘explosive’ stomach illness exceeds 1,000 cases in northern state
NEWYou can now listen to Fox News articles!
Cases of cyclosporiasis infections are rising across America, with more than 1,000 people diagnosed in Michigan and more than 500 in Ohio.
This is the largest outbreak of its kind in Michigan’s history and one of the country’s largest in years, according to the Associated Press.
The parasitic infection can cause weeks of watery diarrhea. The source of the infections has not been identified and no deaths have been reported.
MYSTERY PARASITE LEAVES AMERICANS BATTLING ‘EXPLOSIVE’ ILLNESS AS CDC INVESTIGATES
Michigan officials announced the outbreak last week following the identification of more than 170 cases since June 22 in the southeastern part of the state. Typically, only about 50 cases are identified in Michigan each year, according to AP.
A cyclospora infection often causes watery, “explosive” diarrhea that can last for weeks or even months if left untreated, the CDC says. (iStock)
Similar illnesses have been reported in 28 other states, including neighboring Ohio, where diagnoses have popped up across the Michigan border.
Cases have been climbing since the CDC identified an uptick in infections in mid-June, with illnesses now reported in dozens of states.
CLICK HERE TO SIGN UP FOR OUR HEALTH NEWSLETTER
Michigan has now reported more than 1,200 cases and at least 40 hospitalizations. In Ohio, northwest counties have identified more than 500 cases, including at least 306 in Lucas County, according to the latest available local figures.
TEST YOURSELF WITH OUR LATEST LIFESTYLE QUIZ
Texas has reported at least 48 cases, and illnesses have been identified in numerous other states as health officials investigate the source.
The hallmark symptom of a cyclospora infection is watery, often “explosive” diarrhea that can last for weeks or even months if left untreated, the CDC says.
Other symptoms of cyclosporiasis include severe abdominal cramping, bloating, nausea, fatigue and significant weight loss. (iStock)
Other symptoms include severe abdominal cramping, bloating, nausea, fatigue and significant weight loss.
The official outbreak season for the parasite runs from May 1 through Aug. 31, a window where warmer temperatures historically coincide with a spike in infections, according to the CDC.
CLICK HERE FOR MORE HEALTH STORIES
While the infection can sometimes clear up on its own, it frequently requires antibiotics. The CDC advises anyone experiencing symptoms of cyclosporiasis to contact a healthcare provider for testing and treatment.
The CDC, alongside the Food and Drug Administration and state health officials, is actively investigating several multistate clusters, but they have yet to find a cause behind the spread.
Fresh produce should be washed thoroughly before eating, although this may not eliminate the risk of infection, the AP noted. (iStock)
Past infections have reportedly been linked to consuming contamined fruits or vegetables, or being exposed to contaminated irrigation water.
Fresh produce should be washed thoroughly before being eaten, although this may not eliminate the risk of infection, the AP noted.
CLICK HERE TO DOWNLOAD THE FOX NEWS APP
Michigan officials recommend purchasing salad mixes or whole heads of lettuce instead of pre-washed, bagged lettuce. Remove two to three leaves from the outer layer of the lettuce head before washing and cook vegetables when possible.
Fox News Digital’s Khloe Quill contributed to this report.
NEWYou can now listen to Fox News articles!
Washington, DC – GLP-1 weight-loss drugs have become a prevalent part of American healthcare, and the current administration is getting behind the movement.
In an exclusive interview with Fox News Digital at the Great American State Fair in the nation’s capital on July 6, CMS Administrator Dr. Mehmet Oz voiced his support for the use of GLP-1 (glucagon-like peptide-1) medications, such as Ozempic, for appropriate uses.
“I’m a fan of GLP-1 drugs when used correctly,” he said. “They do help people who are overweight lose weight quite effectively. They’re not a replacement for diet and exercise, but they might jumpstart the system so it’s easier for you to use healthier tactics.”
DR OZ WARNS MEDICARE SCAMMERS ARE STEALING BILLIONS — AND YOUR PERSONAL INFORMATION COULD BE NEXT
This is especially helpful for those who may have trouble moving due to joint pain or are experiencing internal dysfunction, Oz said.
Certain GLP-1 drugs are covered by Medicare for overweight candidates with certain conditions, such as high blood pressure and diabetes, and Oz projected the benefits will continue to benefit taxpayers.
Dr. Mehmet Oz is pictured in Washington, D.C., at the Great American State Fair, where he spoke about federal health policy. (Angelica Stabile/Fox News Digital)
“We believe these are so effective in reducing conditions like high blood pressure and diabetes that they’ll actually save money for the federal taxpayer, because [they’re] going to make you healthy enough that you don’t have to consume health services,” Oz said.
CLICK HERE TO SIGN UP FOR OUR HEALTH NEWSLETTER
“We think about 70% of all the money we spend on healthcare is caused by chronic conditions, and obesity is the No. 1 driver of all that, so it’s a smart decision.”
Oz recently announced the launch of the Medicare GLP-1 Bridge program, which will allow more seniors to access GLP-1 drugs for only $50 a month if they meet qualifying health criteria and receive prior authorization from a doctor.
The doctor expressed support for broadening affordable access to GLP-1 medications for Americans. (iStock)
“There are a lot of overweight people who don’t have high blood pressure, diabetes or other conditions, so they don’t get access to the drug normally,” he said. “We want them to have the ability to use it as well.”
Although these access shifts could boost Americans’ overall health — and in some cases could be lifesaving — Oz noted that there is “no silver bullet” when it comes to these medications.
CLICK HERE FOR MORE HEALTH STORIES
“I love the fact that the innovation that’s coming out of pharmaceutical companies is allowing us to save lives and make lives better,” he said. “But the real secret to longevity is eating right, exercising, sleeping, dealing with the stress of your life, finding some purpose in your existence [and] realizing you have agency over the future.”
“These are things that your mom would have told you [and that] you don’t need a doctor to be emphasizing.”
Medicating appropriately, combined with eating right, exercising and staying connected with others, can help make health goals attainable, the doctor said. (iStock)
While GLP-1s may not be a fix-all, combining these medications with foundational health practices “makes a lot of sense,” Oz said.
CLICK HERE TO DOWNLOAD THE FOX NEWS APP
“I don’t want people being fat-shamed … I don’t want you feeling guilty that you’re gaining weight even though everyone else around you seems to have figured it out,” he said. “It’s not that simple — our set points for hunger are different. We have different things going on in our lives.”
TEST YOURSELF WITH OUR LATEST LIFESTYLE QUIZ
“But if you realize how precious you are — the temple of the soul is so valuable. It’s the greatest gift your parents ever gave you, and you take advantage of every tool out there to make it work … which includes using medications when appropriate. That, to me, is MAHA.”
-
Health15 minutes ago3 Best Peptides for Weight Loss—and the Viral One Doctors Say To Skip
-
Lifestyle30 minutes agoSmithsonian chief emphasizes ‘accuracy and integrity’ after White House report
-
Technology42 minutes agoMicrosoft’s carbon emissions went up 25 percent last year
-
World45 minutes agoJailed Catholic woman’s hunger strike highlights Iran religious persecution — US demands action
-
Politics50 minutes agoIsrael shares intelligence warning Iran plotted new assassination attempt against Trump: report
-
Health57 minutes agoParasitic infection causing ‘explosive’ stomach illness exceeds 1,000 cases in northern state
-
Sports1 hour agoLondon descends into disorder as Morocco fans flood streets after World Cup elimination by France
-
Technology1 hour agoGoogle turns old phones into cloud servers
