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THERAPEUTIC STRATEGIES TO TREAT CNS PATHOLOGY IN MUCOPOLYSACCHARIDOSES
THERAPEUTIC STRATEGIES TO TREAT CNS PATHOLOGY IN MUCOPOLYSACCHARIDOSES
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机译:治疗策略在粘多糖中治疗中枢神经系统病理
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摘要
The invention provides for nucleotide sequences encoding for a chimeric sulfatase, viral vectors expressing such sequences for gene therapy and pharmaceutical uses of the chimeric expressed protein. The invention is particularly applied in the therapy of mucopolysaccharidosis, preferably type IIIA.
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