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New gene therapy could significantly reduce seizures in severe childhood epilepsy
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New gene therapy could significantly reduce seizures in severe childhood epilepsy

by University College London CAMK2A-EKC therapy reduces seizure frequency. A. Timeline. B. Heatmap displaying seizure occurrence in each animal injected with either CAMK2A-GFP (gray) or CAMK2A-EKC virus (purple) before and after the treatment (virus injection on day 0). Crosses correspond to days where data acquisition was incomplete. C. Graph displaying the change in number of seizures...

FDA advisors say new gene therapy for sickle cell disease is safe
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FDA advisors say new gene therapy for sickle cell disease is safe

by Physician’s Briefing Staff A new gene therapy for sickle cell disease has been deemed safe by a U.S. Food and Drug Administration advisory panel, paving the way for full approval by early December 2023. The FDA had already decided that the therapy, known as exagamglogene autotemcel (exa-cel), was effective. Developed by Vertex Pharmaceuticals of...

New gene therapy could prevent genetic hearing loss
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New gene therapy could prevent genetic hearing loss

By Michael Irving August 11, 2022 A new gene therapy could correct forms of genetic hearing loss Depositphotos Researchers at the Salk Institute have made a breakthrough that could lead to new treatments for genetic hearing loss. Gene therapy that delivers a particular protein can ensure faulty hair cells grow correctly, allowing for improved hearing....