gene therapy
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Developing effective treatments for genetic lung diseases such as cystic fibrosis has proven challenging. That might not be the case for much longer, with scientists developing a new type of nanoparticle that can carry gene-editing technology directly into the lungs of mice.
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Scientists have identified 238 genes involved in Alzheimer's disease, and one in particular piqued their interest. They believe the Surf4 gene's role in keeping messaging pathways open offers new clues as to how this devastating disease takes hold.
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Using a refined version of the CRISPR gene-editing tool, researchers have restored vision in mice with retinitis pigmentosa. The results may open the door for treatments for the one in 5,000 humans afflicted with the blindness-inducing condition.
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It’s long been thought that migraine and blood sugar level issues have been related, but now scientists have identified that they're genetically linked, opening the door for novel therapies and even prevention strategies for sufferers.
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ALS is a debilitating neurodegenerative disease with poor outcomes, but a pair of new studies may point towards a more optimistic future. In tests in human cells and live mice, scientists identified promising new targets for potential treatment.
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Researchers studying healthy centenarians have homed in on a gene mutation that plays a crucial role in maintaining cardiovascular health in old age. Across a series of experiments the researchers demonstrated how gene therapy could rejuvenate aging hearts.
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Harvard scientists have investigated why we age, and how we could stop it. In tests in mice, the team showed that epigenetic “software glitches” drive the symptoms of aging – and a system reboot can reverse them, potentially extending lifespan.
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It's often hard to see the forest from the trees but if we zoom out a little it becomes clear 2022 saw some incredible breakthroughs in the world of medical science. All of these stories are landmark moments that are set to shape the future of medicine.
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A rare genetic disease that renders children without a functioning immune system from birth has been effectively cured by an experimental gene therapy. A study has reported on the first 10 children treated with the therapy, all of whom are now healthy.
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A Phase 3 trial testing the world's first topical gene therapy has reported extraordinary results in children suffering from a rare blistering skin disease. The gene therapy gel was seen to completely heal wounds that had been open for years.
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A new technique has been added to the CRISPR gene-editing toolbox. Known as PASTE, the system uses virus enzymes to “drag-and-drop” large sections of DNA into a genome, which could help treat a range of genetic diseases.
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The US Food and Drug Administration (FDA) has approved the first ever cell-based gene therapy, a one-off treatment for people with a rare genetic blood disease. The treatment will cost US$2.8 million, making it the most expensive medicine in history.
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