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Scientists lay foundation for potential gene-editing therapy for late-onset Tay-Sachs disease

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  • 2025-08-18 18:37 event
  • 7 hours ago schedule
Scientists lay foundation for potential gene-editing therapy for late-onset Tay-Sachs disease
Scientists at the National Institutes of Health (NIH) have successfully reduced the severity of late-onset Tay-Sachs (LOTS) disease in human cell cultures and a mouse model by using a novel gene-editing treatment.

22. New screener offers empirical insights to improve veteran transitions

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For the newest generation of U.S. veterans, transitioning to civilian life can pose profound challenges. Post-9/11 veterans are younger and more diverse, and they face rising rates of trauma, mental health conditions, underemployment and service-connected disabilities, according to a research team at Penn State. To help veterans tackle these hurdles, the researchers created an online assessment tool that assists veteran-serving organizations identify individualized risks and provide targeted, evidence-informed support.

23. 1 in 3 Australian e-scooter fatalities are children

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Recently, I've repeatedly come across tragic news reports of children killed in electric scooter, or e-scooter, crashes.

24. DNA repair enzyme Polβ shields developing brain from harmful mutations, study reveals

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A research group led by the University of Osaka has discovered that the DNA repair enzyme Polβ plays a crucial role in protecting the developing brain from harmful mutations. The study found that a lack of Polβ leads to a significant increase in small insertions and deletions of DNA, known as indels near CpG sites, which are important regulatory regions in genes. This accumulation of mutations could contribute to neurodevelopmental disorders.

25. 'Clear' e-cigarettes linked to greater acute rises in blood pressure and heart rate

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Department of Chemical and Environmental Engineering, Yale University and Boston University Chobanian, Avedisian School of Medicine-led researchers report that e-cigarettes labeled "clear" produced greater acute increases in blood pressure and heart rate than non "clear" flavored products or nonuse, and that "clear" e-liquids contained synthetic cooling agents alongside menthol and other flavorants.

26. Doctors seek best way to treat newly discovered genetic disorder

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Most folks probably think of gene mutations as something people are born with, but genes can also change later in life, sometimes making people sick.

27. Early lead exposure could result in memory issues later in life

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A new study has found that people who lived in areas with high levels of leaded gasoline emissions in the 1960s and '70s are more likely to report memory problems today—a finding that researchers say could deepen our understanding of environmental risks tied to dementia.

28. Patient numbers at NIH hospital have plummeted under Trump, jeopardizing care

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The number of people receiving treatment at the National Institutes of Health Clinical Center—the renowned research hospital that cares for patients with rare or life-threatening diseases—has tumbled under the second Trump administration, according to government documents and interviews with current and former NIH employees.

29. Researchers uncover potential mechanism driving treatment resistance in common breast cancer

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A team of scientists at the Garvan Institute of Medical Research has discovered that inactivation of a stress pathway makes ER+ breast cancer cells ignore stress signals, allowing them to evade treatment.

30. Kratom faces increasing scrutiny from states and the feds

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For years, state lawmakers have taken the lead on regulating kratom—the controversial herbal supplement used for pain relief, anxiety and opioid withdrawal symptoms. Some states have banned it entirely. Others have passed laws requiring age limits, labeling and lab testing.

31. Scientists lay foundation for potential gene-editing therapy for late-onset Tay-Sachs disease

  • 7 hours ago schedule
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Scientists at the National Institutes of Health (NIH) have successfully reduced the severity of late-onset Tay-Sachs (LOTS) disease in human cell cultures and a mouse model by using a novel gene-editing treatment.

32. Amblyopia (lazy eye) treatment: Eye patching, drops, digital therapies and more

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Amblyopia, also known as "lazy eye," is a common eye condition in kids that develops when a child has an eye that does not see well. This is caused by a poor connection between the eye to the brain, which can happen for a variety of different reasons. Amblyopia is more common in one eye but can also happen in both eyes.

33. COVID-19 surges nationwide with highest rates in Southwest as students return to school

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COVID-19 rates in the Southwestern United States reached 12.5%—the highest in the nation—according to new data from the U.S. Centers for Disease Control and Prevention. Meanwhile, Los Angeles County recorded the highest COVID-19 levels in its wastewater since February.

34. Harnessing VR to prevent substance use relapse

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Substance use recovery is a lifelong process, but environmental triggers, such as alcohol at social gatherings or pain medication advertisements, can put individuals in recovery at risk of relapse.

35. COVID-19 cases surge in Maryland as summer ends and school begins

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COVID-19 hospitalizations in Maryland are up 123% over the last two weeks, the most hospitalizations in a month since April. While the state has typically seen a surge in COVID-19 cases during summer, this one is coming later than usual.

36. California valley fever cases hit record highs again in 2025, state reports

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The latest California numbers suggest 2025 will be another record-smashing year for valley fever, the illness linked to drought and precipitation and spread by fungal spores.

37. Prioritize sleep as the school year begins, say experts

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As the new school year begins, experts say one simple habit can help kids succeed: getting enough sleep.

38. An alternative to LASIK—without the lasers

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Millions of Americans have altered vision, ranging from blurriness to blindness. But not everyone wants to wear prescription glasses or contact lenses. Accordingly, hundreds of thousands of people undergo corrective eye surgery each year, including LASIK—a laser-assisted surgery that reshapes the cornea and corrects vision.

39. Small molecule could alleviate acetaminophen-induced liver injury

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Acetaminophen is one of the most common painkillers and is found in hundreds of different medications. While safe at recommended doses, acetaminophen overdose is the leading cause of acute liver injury in the U.S.

40. Ketamine use in chronic pain unsupported by evidence, review finds

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The off-label use of ketamine to treat chronic pain is not supported by scientific evidence, a new Cochrane review has found.

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