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Home » AAV to deliver SynGAP1 gene against neurological disorders
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Neurology/psychiatric

AAV to deliver SynGAP1 gene against neurological disorders

Oct. 15, 2025
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Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
BioWorld Science Genetic/congenital Neurology/psychiatric Gene therapy

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