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Scientists Edited Genes Inside a Living Person for the First Time—and Saved His Life
Scientists Edited Genes Inside a Living Person for the First Time—and Saved His Life

Yahoo

time20-05-2025

  • Health
  • Yahoo

Scientists Edited Genes Inside a Living Person for the First Time—and Saved His Life

"Hearst Magazines and Yahoo may earn commission or revenue on some items through these links." Here's what you'll learn in this story. The world's first bespoke gene therapy saved the life of a newborn with a rare genetic disorder that cause the build-up of life-threatening ammonia in the body. In a race against time, scientists and doctors across the U.S. developed the first in vivo gene therapy, thanks to decades of medical research. After three doses, the newborn patient showed drastic improvement, and this new era of in vivo gene therapies could save the lives of millions more in the future. Life's ability to successfully copy three billion distinct letters in the human genome is an absolute biological wonder—but sometimes, mistakes are made. Whether inherited or formed in utero, genetic disorders and other birth defects are common, and occur in one in every 33 babies in the U.S., according to the Centers of Disease Control and Prevention (CDC). For all of human history, a person born with such a disorder likely had to live with the condition, and depending on the defect, those lives could be brutally short. But in 2025, human history changed forever. In a groundbreaking announcement, detailed in a study published in the New England Journal of Medicine, scientists, doctors, and specialists from institutions around the U.S.—including the Children's Hospital of Philadelphia, University of California-Berkeley, and Penn Medicine—successfully saved the life of a newborn patient named KJ, who had been born with a rare genetic disorder. To pull off this incredible medical feat, doctors employed the world's first custom in vivo (i.e. inside a living organism, rather than in a petri dish) CRISPR gene therapy. This technique, developed over decades thanks to U.S.-funded medical research, could help alleviate painful lives for millions of people born every year with now-fixable genetic disorders. 'Years and years of progress in gene editing and collaboration between researchers and clinicians made this moment possible, and while KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient's needs,' Children's Hospital of Philadelphia's Rebecca Ahrens-Nicklas, a co-author of the study, said in a press statement. The details of this incredible medical intervention play out like a made-for-tv medical drama, but the stakes were incredibly real and deadly serious. A week after his birth, doctors noticed something wasn't quite right with KJ. After ruling out a few possibilities, they stumbled across the unfortunate answer—a rare genetic disorder called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency that affects only one in every 1.3 million babies. This disorder inhibits the body's ability to get rid of ammonia, a product of protein metabolism. This can have deadly consequences, impact brain development, and wreak havoc on the liver. Usually, the treatment for a disorder like this is a liver transplant, but that was not an option for the infant boy, who was still too young to be considered for the surgery. So, once arriving at a diagnosis, Ahrens-Nicklas contacted a gene-editing specialist at the University of Pennsylvania named Kiran Musunuru and 'the clock start[ed]in my mind,' he later told The New York Times. Working with a team of specialists across the country for six months, Ahrens-Nicklas and Musunuru developed a targeted gene therapy to fix KJ's specific variant of CPS1. Meanwhile, KJ was kept under medical surveillance at the hospital and subsisted on a diet completely devoid of protein to avoid making his condition worse. By the time the CRISPR treatment was ready, KJ was in the 7th percentile for his weight. On February 25, the team began administering the treatment, with Ahrens-Nicklas and Musunuru describing the process as both exciting and terrifying. 'One of the most terrifying moments was when I walked into the room and said, 'I don't know if it will work but I promise I will do everything I can to make sure it is safe,'' Ahrens-Nicklas told The New York Times. The first infusion took two hours, and within two weeks, KJ began eating protein like a healthy baby. A second dose arrived 22 days later, and about two weeks ago, KJ received a third. Although it's unknown if he will eventually still need a liver transplant, doctors can now safely say that a human life has been saved thanks to the world's first bespoke in vivo gene therapy—a huge testament to decades of a research and experimentation. KJ is now at home with his family. 'We want each and every patient to have the potential to experience the same results we saw in this first patient, and we hope that other academic investigators will replicate this method for many rare diseases and give many patients a fair shot at living a healthy life,' Musunuru said in a press statement. 'The promise of gene therapy that we've heard about for decades is coming to fruition, and it's going to utterly transform the way we approach medicine.' You Might Also Like The Do's and Don'ts of Using Painter's Tape The Best Portable BBQ Grills for Cooking Anywhere Can a Smart Watch Prolong Your Life?

241 passengers, crew sickened with norovirus on luxury cruise ship
241 passengers, crew sickened with norovirus on luxury cruise ship

Yahoo

time02-04-2025

  • Health
  • Yahoo

241 passengers, crew sickened with norovirus on luxury cruise ship

More than 200 luxury cruise ship passengers caught norovirus on a monthlong transatlantic voyage that won't officially end until Sunday, U.S. health officials said. A new outbreak report from the Centers of Disease Control and Prevention tracked 224 of 2,538 passengers who became ill from the virus while on board the Cunard cruise line ship Queen Mary 2, along with 17 crew members. The vessel carried 1,232 crew overall, according to the CDC. Queen Mary 2 left Southampton in the United Kingdom March 8 on a route that first took it to New York and, then, down through the Caribbean, making various stops along the island chain, including St. Thomas and Barbados, according to an itinerary posted online by Cunard. The cruise sailed back to the U.K. on the same course it took to get to the Caribbean and is due to return to Southampton on April 6. Passengers and crew who contracted norovirus on the Queen Mary 2 mainly experienced diarrhea and vomiting, two of the most common symptoms of the highly contagious bug. The outbreak was reported to the CDC's vessel sanitation program, which monitors cruise ship outbreaks remotely and helps guide how crews respond to them, on March 18. The CDC said the crew of Queen Mary 2 worked to contain the spread of the virus by increasing cleaning and disinfection procedures, conducting tests to confirm cases of the illness and isolating sick passengers and crew. This is the latest in a series of norovirus outbreaks on ocean liners operated by some of the world's largest cruise companies. The latest outbreak comes on the heels of a surge in confirmed cases around the U.S. toward the end of last year. Also known as a stomach bug, norovirus is a very contagious disease that usually causes symptoms for 1 to 3 days, although health officials say people can still transmit the virus to others for two weeks or longer after their own symptoms clear up. In addition to Queen Mary 2, at least nine cruise ships have reported outbreaks since the start of 2025, according to the CDC. That tally includes voyages run by major cruise companies like Holland America and Princess Cruises, among others. CBS News contacted Cunard for comment but did not receive an immediate reply. Democratic-backed candidate wins record-breaking Wisconsin Supreme Court seat Eric Adams corruption case dismissed with prejudice Blue Origin's all-female space crew featured on special digital cover of Elle Magazine

241 passengers, crew sickened with norovirus on luxury cruise ship
241 passengers, crew sickened with norovirus on luxury cruise ship

CBS News

time02-04-2025

  • Health
  • CBS News

241 passengers, crew sickened with norovirus on luxury cruise ship

More than 200 luxury cruise ship passengers caught norovirus on a monthlong transatlantic voyage that won't officially end until Sunday, U.S. health officials said. A new outbreak report from the Centers of Disease Control and Prevention tracked 224 of 2,538 passengers who became ill from the virus while on board the Cunard cruise line ship Queen Mary 2, along with 17 crew members. The vessel carried 1,232 crew overall, according to the CDC. Queen Mary 2 left Southampton in the United Kingdom March 8 on a route that first took it to New York and, then, down through the Caribbean, making various stops along the island chain, including St. Thomas and Barbados, according to an itinerary posted online by Cunard. The cruise sailed back to the U.K. on the same course it took to get to the Caribbean and is due to return to Southampton on April 6. Passengers and crew who contracted norovirus on the Queen Mary 2 mainly experienced diarrhea and vomiting, two of the most common symptoms of the highly contagious bug. The outbreak was reported to the CDC's vessel sanitation program, which monitors cruise ship outbreaks remotely and helps guide how crews respond to them, on March 18. The CDC said the crew of Queen Mary 2 worked to contain the spread of the virus by increasing cleaning and disinfection procedures, conducting tests to confirm cases of the illness and isolating sick passengers and crew. This is the latest in a series of norovirus outbreaks on ocean liners operated by some of the world's largest cruise companies. The latest outbreak comes on the heels of a surge in confirmed cases around the U.S. toward the end of last year. Also known as a stomach bug, norovirus is a very contagious disease that usually causes symptoms for 1 to 3 days, although health officials say people can still transmit the virus to others for two weeks or longer after their own symptoms clear up. In addition to Queen Mary 2, at least nine cruise ships have reported outbreaks since the start of 2025, according to the CDC . That tally includes voyages run by major cruise companies like Holland America and Princess Cruises, among others. CBS News contacted Cunard for comment but did not receive an immediate reply.

'A continual cycle': Losing a baby late in pregnancy is more common in the South, new report shows
'A continual cycle': Losing a baby late in pregnancy is more common in the South, new report shows

Yahoo

time06-03-2025

  • Health
  • Yahoo

'A continual cycle': Losing a baby late in pregnancy is more common in the South, new report shows

Losing a baby late in pregnancy is more common in the South than in other regions of the United States, according to a new report given exclusively to NBC News. The difference is dramatic: Compared with other parts of the country, the odds of having a high rate of late-stage pregnancy loss are nearly three times greater in Southern states. The lack of Medicaid expansion in the South, along with the region's large rural population and relatively low levels of adequate prenatal care, contributes to the high rates of fetal loss in the second half of pregnancy, according to the report by United States of Care, a nonpartisan health care advocacy organization. 'We are losing lives,' said Venice Haynes, lead author of the report. 'At what point are we going to stand up and say enough is enough?' The new analysis found that the mean fetal mortality rate for 16 Southern states in 2023 was 6.05 fetal deaths at 20 weeks' gestation or more per 1,000 live births and fetal deaths, compared with a mean fetal mortality rate of 5.25 for all other states. The analysis is based on provisional data from the Centers of Disease Control and Prevention. Fetal deaths after 24 weeks' gestation have been declining steadily since 1990, according to a CDC report last year. States in the South — including Mississippi, Georgia and Arkansas — had the highest rates. The new report found that the South also has higher rates of postpartum depression and fewer perinatal health workers compared to other regions in the United States. The new data is 'a very significant finding,' said Dr. Amanda Williams, interim chief medical officer of the March of Dimes, which has found that the South also has the highest rates of preterm births in the country. Williams said she isn't optimistic about the outlook for mothers and babies in the South. Congress is considering drastic cuts to Medicaid. Nationally Medicaid covers 41% of births, with especially high rates in many Southern states. In addition, nearly every Southern state has a strict abortion ban, and research suggests such laws are prompting obstetricians to leave and discouraging young doctors from working there. 'We anticipate it will get worse,' Williams said. 'This is an area of the country that needs more investment, more support, more providers, not less.' There are many causes of late stage pregnancy loss — or stillbirths — including infection or a fetus' genetic condition. Often the cause is never known. One reason for the high stillbirth rate in the South is that Black women experience fetal loss at more than twice the rate of other groups. Black women have higher rates of underlying diseases such as hypertension and diabetes, which can put a pregnancy at risk. More than half of the Black population lives in the South, according to a recent Pew Research report. Black women also have high rates of stress, which can affect a pregnancy. Kim Smith, a Black woman who lives in South Carolina, developed a severe type of preeclampsia called HELLP syndrome, and when she was five months pregnant she lost her daughter, Lauren Kelly. Some studies have shown a possible link between stress and preeclampsia, a condition in which a pregnant woman develops dangerously high blood pressure. Smith, 42, was raised in Greenville, North Carolina, by a single mother. 'My mother's a minister, my aunts are ministers, my uncles are bishops. We grew up in a very loving and faithful family,' she said. But Smith's family was poor and lived in a neighborhood with frequent shootings and high rates of drug abuse. Racial tensions were high: She remembers as a child hearing people making racist comments, and the Ku Klux Klan sometimes marched near her neighborhood. 'I recall as a child hearing gunshots and hearing police sirens and ambulance sirens on a regular basis,' she said. Smith married and moved to Irmo, South Carolina. When she became pregnant with her daughter in 2011, she was an administrator at federally qualified health care centers in underprivileged neighborhoods with poor health outcomes. It was difficult work as she observed the impact of poverty and racism on the community. 'That level of stress is not unique to me. It was just kind of the culture,' she said. While she worked hard and relied on her faith and her family's love, she said, it wasn't always easy. 'When you experience life, what do you do? You make the best of it, and you push through. And we give ourselves a badge for that. But at the end of the day, our bodies remember the stress,' she said. 'Your body remembers what you experience.' After she lost her baby, Smith changed jobs and took other steps to reduce stress in her life. She went on to have three sons — all healthy pregnancies — and is now a health tech entrepreneur. In many ways, the South is a perfect storm for bad outcomes for pregnant women, according to an analysis done for NBC News by the Perinatal Data Center at the March of Dimes. Compared with other parts of the country, the South has the highest percentage of uninsured women ages 19-54 and the highest percentage of women that age living in maternity care deserts, where access to maternity care is limited or absent, according to the March of Dimes. The region also has the highest percentage of births in which the mothers received inadequate prenatal care. Even though the South stands out, the entire United States suffers from high rates of pregnancy and postpartum complications, said Natalie Davis, CEO of United States of Care. The maternal mortality rate in the United States is higher than in other developed countries — more than four times the rates in the United Kingdom, Australia or Germany, according to the Commonwealth Fund. The group has also found that the United States has a low supply of midwives and OB-GYNs compared with most other developed countries — 16 per 1,000 live births compared with, for example, 35 in the Netherlands and 43 in France per 1,000 live births. 'Our message to every governor is that no state's doing well at this,' she said. 'Every state needs to focus on this.' There are high-tech and low-tech approaches to addressing pregnancy complications like stillbirth. Researchers are developing artificial intelligence tools to predict which pregnant women are likely to go into premature labor. Programs such as Kid One in Alabama and Green Cars for Kids in Florida provide rides for expectant mothers to get to their prenatal appointments. 'They're missing their appointments because bus connections are too long or there isn't even a bus that goes to their neighborhood,' said Dr. Catherine Tom, president of Green Cars for Kids. Providing the rides so the women can get care 'is so simple — it's not rocket science.' Another program, Nurse-Family Partnership, connects low-income first-time mothers-to-be in 40 states with nurses. Sharon Sprinkle, the director of the program in several Southern states, said her teams have prevented many 'near misses' by educating their clients about signs of early labor, preeclampsia and other pregnancy complications. Too often, pregnant women report back that their concerns were 'minimized or totally dismissed, when, in fact, if they were listened to, the outcome would have been better,' she said. Nurses in the program teach clients to 'unleash their power and be comfortable having conversations with physicians to say: 'This is what's going on. I need you to listen. And if you won't listen, find somebody else in your office who will listen.'' Sprinkle, a registered nurse who has worked in infant and maternity care in the South for decades, said she isn't surprised by the new findings about stillbirths. Neither is Dr. Cornelia Graves, a maternal-fetal medicine specialist and professor at the University of Tennessee Health Science Center. Graves said poor pregnancy outcomes in the South will continue because of high rates of underlying diseases such as diabetes, obesity and heart disease. 'We know that women who are unhealthy give birth to unhealthy babies who then become unhealthy children who then become unhealthy mothers,' she said. 'It is a continual cycle.' This article was originally published on

Late pregnancy loss more common in the South
Late pregnancy loss more common in the South

NBC News

time06-03-2025

  • Health
  • NBC News

Late pregnancy loss more common in the South

Losing a baby late in pregnancy is more common in the South than in other regions of the United States, according to a new report given exclusively to NBC News. The difference is dramatic: Compared with other parts of the country, the odds of having a high rate of late-stage pregnancy loss are nearly three times greater in Southern states. The lack of Medicaid expansion in the South, along with the region's large rural population and relatively low levels of adequate prenatal care, contributes to the high rates of fetal loss in the second half of pregnancy, according to the report by United States of Care, a nonpartisan health care advocacy organization. 'We are losing lives,' said Venice Haynes, lead author of the report. 'At what point are we going to stand up and say enough is enough?' The new analysis found that the mean fetal mortality rate for 16 Southern states in 2023 was 6.05 fetal deaths at 20 weeks' gestation or more per 1,000 live births and fetal deaths, compared with a mean fetal mortality rate of 5.25 for all other states. The analysis is based on provisional data from the Centers of Disease Control and Prevention. Fetal deaths after 24 weeks' gestation have been declining steadily since 1990, according to a CDC report last year. S tates in t he South — including Mississippi, Georgia and Arkansas — had the highest rates. The new report found that the South also has higher rates of postpartum depression and fewer perinatal health workers compared to other regions in the United States. The new data is 'a very significant finding,' said Dr. Amanda Williams, interim chief medical officer of the March of Dimes, which has found that the South also has the highest rates of preterm births in the country. Williams said she isn't optimistic about the outlook for mothers and babies in the South. Congress is considering drastic cuts to Medicaid. Nationally Medicaid covers 41% of births, with especially high rates in many Southern states. In addition, nearly every Southern state has a strict abortion ban, and research suggests such laws are prompting obstetricians to leave and discouraging young doctors from working there. 'We anticipate it will get worse,' Williams said. 'This is an area of the country that needs more investment, more support, more providers, not less.' 'Your body remembers' There are many causes of late stage pregnancy loss — or stillbirths — including infection or a fetus' genetic condition. Often the cause is never known. One reason for the high stillbirth rate in the South is that Black women experience fetal loss at more than twice the rate of other groups. Black women have higher rates of underlying diseases such as hypertension and diabetes, which can put a pregnancy at risk. More than half of the Black population lives in the South, according to a recent Pew Research report. Black women also have high rates of stress, which can affect a pregnancy. Kim Smith, a Black woman who lives in South Carolina, developed a severe type of preeclampsia called HELLP syndrome, and when she was five months pregnant she lost her daughter, Lauren Kelly. Some studies have shown a possible link between stress and preeclampsia, a condition in which a pregnant woman develops dangerously high blood pressure. Smith, 42, was raised in Greenville, North Carolina, by a single mother. 'My mother's a minister, my aunts are ministers, my uncles are bishops. We grew up in a very loving and faithful family,' she said. But Smith's family was poor and lived in a neighborhood with frequent shootings and high rates of drug abuse. Racial tensions were high: She remembers as a child hearing people making racist comments, and the Ku Klux Klan sometimes marched near her neighborhood. 'I recall as a child hearing gunshots and hearing police sirens and ambulance sirens on a regular basis,' she said. Smith married and moved to Irmo, South Carolina. When she became pregnant with her daughter in 2011, she was an administrator at federally qualified health care centers in underprivileged neighborhoods with poor health outcomes. It was difficult work as she observed the impact of poverty and racism on the community. 'That level of stress is not unique to me. It was just kind of the culture,' she said. While she worked hard and relied on her faith and her family's love, she said, it wasn't always easy. 'When you experience life, what do you do? You make the best of it, and you push through. And we give ourselves a badge for that. But at the end of the day, our bodies remember the stress,' she said. 'Your body remembers what you experience.' After she lost her baby, Smith changed jobs and took other steps to reduce stress in her life. She went on to have three sons — all healthy pregnancies — and is now a health tech entrepreneur. 'No state's doing well' In many ways, the South is a perfect storm for bad outcomes for pregnant women, according to an analysis done for NBC News by the Perinatal Data Center at the March of Dimes. Compared with other parts of the country, the South has the highest percentage of uninsured women ages 19-54 and the highest percentage of women that age living in maternity care deserts, where access to maternity care is limited or absent, according to the March of Dimes. The region also has the highest percentage of births in which the mothers received inadequate prenatal care. Even though the South stands out, the entire United States suffers from high rates of pregnancy and postpartum complications, said Natalie Davis, CEO of United States of Care. The maternal mortality rate in the United States is higher than in other developed countries — more than four times the rates in the United Kingdom, Australia or Germany, according to the Commonwealth Fund. The group has also found that the United States has a low supply of midwives and OB-GYNs compared with most other developed countries — 16 per 1,000 live births compared with, for example, 35 in the Netherlands and 43 in France per 1,000 live births. ' Our message to every governor is that no state's doing well at this,' she said. 'Every state needs to focus on this.' Stopping a 'continual cycle' in the South There are high-tech and low-tech approaches to addressing pregnancy complications like stillbirth. Researchers are developing artificial intelligence tools to predict which pregnant women are likely to go into premature labor. Programs such as Kid One in Alabama and Green Cars for Kids in Florida provide rides for expectant mothers to get to their prenatal appointments. 'They're missing their appointments because bus connections are too long or there isn't even a bus that goes to their neighborhood,' said Dr. Catherine Tom, president of Green Cars for Kids. Providing the rides so the women can get care 'is so simple — it's not rocket science.' Another program, Nurse-Family Partnership, connects low-income first-time mothers-to-be in 40 states with nurses. Sharon Sprinkle, the director of the program in several Southern states, said her teams have prevented many 'near misses' by educating their clients about signs of early labor, preeclampsia and other pregnancy complications. Too often, pregnant women report back that their concerns were 'minimized or totally dismissed, when, in fact, if they were listened to, the outcome would have been better,' she said. Nurses in the program teach clients to 'unleash their power and be comfortable having conversations with physicians to say: 'This is what's going on. I need you to listen. And if you won't listen, find somebody else in your office who will listen.'' Sprinkle, a registered nurse who has worked in infant and maternity care in the South for decades, said she isn't surprised by the new findings about stillbirths. Neither is Dr. Cornelia Graves, a maternal-fetal medicine specialist and professor at the University of Tennessee Health Science Center. Graves said poor pregnancy outcomes in the South will continue because of high rates of underlying diseases such as diabetes, obesity and heart disease. 'We know that women who are unhealthy give birth to unhealthy babies who then become unhealthy children who then become unhealthy mothers,' she said. 'It is a continual cycle.'

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