Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
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This European treatment for joint pain just passed a major scientific test
Korean researchers found that low-dose radiation therapy eased knee pain and improved movement in people with mild to moderate osteoarthritis. The treatment, far weaker than cancer radiation, showed real benefits beyond placebo. With no side effects and strong trial results, the approach could provide a middle ground between painkillers and joint surgery.
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Brain fat, not just plaques, may be the hidden driver of Alzheimer’s
For decades, scientists believed Alzheimer’s was driven mainly by sticky protein plaques and tangles in the brain. Now Purdue researchers have revealed a hidden culprit: fat. They found that brain immune cells can become clogged with fat, leaving them too weak to fight off disease. By clearing out this fat and restoring the cells’ defenses, researchers may have uncovered an entirely new way to combat Alzheimer’s — shifting the focus from plaques alone to how the brain handles fat.
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Scientists finally reveal the hidden mechanism linking alcohol to fatty liver
Mayo Clinic scientists uncovered how excessive drinking triggers fatty liver disease by disrupting the enzyme VCP, which normally prevents harmful protein buildup on fat droplets in the liver. Alcohol blocks this protective process, allowing fat to accumulate and damage liver cells.
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Stanford scientists reveal simple shift that could prevent strokes and obesity nationwide
Switching clocks twice a year disrupts circadian rhythms in ways that harm health. Stanford scientists found permanent standard time would reduce obesity and stroke rates nationwide, making it the strongest option over permanent daylight saving time or seasonal shifts.
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Scientists just made CRISPR three times more effective
Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By wrapping CRISPR’s tools in spherical DNA-coated nanoparticles, researchers tripled gene-editing success rates, improved precision, and dramatically reduced toxicity compared to current methods.
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Scientists just found a tiny molecule that could change how we lose weight
Researchers at the Salk Institute have used CRISPR to uncover hidden microproteins that control fat cell growth and lipid storage, identifying one confirmed target, Adipocyte-smORF-1183. This breakthrough could lead to more effective obesity treatments, surpassing the limitations of current drugs like GLP-1.
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This vaccine uses dental floss instead of needles
Scientists have discovered that flossing between your teeth could one day help vaccinate you. By targeting a uniquely permeable gum tissue called the junctional epithelium, this new method stimulates immunity right where many infections enter: the mouth, nose, and lungs. Using dental floss on mice to apply a flu vaccine triggered a robust immune response—better than existing oral approaches and comparable to nasal vaccines, but without the risks. It even worked with mRNA and protein-based vaccines.
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Breakthrough: How radiation helps the immune system kill cancer
Radiation therapy, once thought of mainly as a local cancer treatment, is now showing power to awaken the immune system in surprising ways. Researchers discovered that combining radiation with immunotherapy can transform stubborn, unresponsive lung tumors into targets for immune attack—especially those considered “cold” and typically resistant. This happens through a rare and poorly understood effect where immune cells are activated systemically, not just at the radiation site. Patients whose tumors underwent this “warm-up” had significantly better outcomes, revealing a promising new strategy for fighting hard-to-treat cancers.
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Three-person DNA IVF stops inherited disease—eight healthy babies born in UK first
In a groundbreaking UK first, eight healthy babies have been born using an IVF technique that includes DNA from three people—two parents and a female donor. The process, known as pronuclear transfer, was designed to prevent the inheritance of devastating mitochondrial diseases passed down through the mother’s DNA. The early results are highly promising: all the babies are developing normally, and the disease-causing mutations are undetectable or present at levels too low to cause harm. For families once haunted by genetic risk, this science offers more than treatment—it offers transformation.
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Iron overload: The hidden culprit behind early Alzheimer’s in Down syndrome
USC researchers have uncovered a hidden driver behind the early and severe onset of Alzheimer's in people with Down syndrome: iron overload in the brain. Their study revealed that individuals with both conditions had twice the iron levels and far more oxidative damage than others. The culprit appears to be ferroptosis, an iron-triggered cell death mechanism, which is especially damaging in sensitive brain regions.
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Scientists design gene delivery systems for cells in the brain and spinal cord
Research teams have created a versatile set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy. These delivery systems are a significant step toward future precise gene therapy to the brain that could safely control errant brain activity with high precision. In contrast, current therapies for brain disorders mostly treat only symptoms.
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CAR-T cell therapy for cancer causes 'brain fog,' study shows
Cancer treatment with a cell-based immunotherapy causes mild cognitive impairment, a Stanford Medicine team found. They also identified compounds that could treat it.
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Researchers map 7,000-year-old genetic mutation that protects against HIV
Modern HIV medicine is based on a common genetic mutation. Now, researchers have traced where and when the mutation arose -- and how it protected our ancestors from ancient diseases.
