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New gene delivery vehicle shows promise for human brain gene therapy

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In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

New gene delivery vehicle shows promise for human brain gene therapy retrieved 16 May 2024 from https://medicalxpress.com/news/2024-05-gene-delivery-vehicle-human-brain.html

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