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Neurodevelopmental disorders

Gene therapy for mucopolysaccharidosis shows promise

Nature Reviews Neurology volume 13, pages 512513 (2017) | Download Citation

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    et al. Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial. Lancet Neurol. (2017)

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