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Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.

著者 Tardieu M , Zérah M , Gougeon ML , Ausseil J , de Bournonville S , Husson B , Zafeiriou D , Parenti G , Bourget P , Poirier B , Furlan V , Artaud C , Baugnon T , Roujeau T , Crystal RG , Meyer C , Deiva K , Heard JM
Lancet Neurol.2017 Jul 13 ; ():.
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Mucopolysaccharidosis type IIIB syndrome (also known as Sanfilippo type B syndrome) is a lysosomal storage disease resulting in progressive deterioration of cognitive acquisition after age 2-4 years. No treatment is available for the neurological manifestations of the disease. We sought to assess the safety and efficacy of a novel intracerebral gene therapy.
PMID: 28713035 [PubMed - as supplied by publisher]
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