gene therapy
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Age-related hearing loss impacts one in three adults aged 64 to 75 in the US, and around half of these cases are down to faulty genes. Using older animals to mirror a human study, scientists successfully reversed hearing loss caused by one such gene.
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A new technique may one day help restore sight to patients with inherited vision impairment. The regenerative therapy works by expressing genes that convert dormant cells into new light-sensing cells in the retina to replace those lost to disease.
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Researchers have developed a highly efficient new gene-editing method that uses virus-based protein fragments. The method could be used to level up existing cell and gene therapies used to treat cancer and other diseases.
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The high eye pressure seen in glaucoma slowly leads to blindness. For some, treatment with eye drops doesn’t work. Researchers have used gene therapy to develop a promising new way of treating the high eye pressure associated with glaucoma.
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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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