about
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Structural insights into the activation mechanism of melibiose permease by sodium binding.Characterization of a recombinant adeno-associated virus type 2 Reference Standard MaterialManufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.Alteration of sugar-induced conformational changes of the melibiose permease by mutating Arg141 in loop 4-5Changes in secondary structures and acidic side chains of melibiose permease upon cosubstrates binding.Studying substrate binding to reconstituted secondary transporters by attenuated total reflection infrared difference spectroscopy.Substrate-induced conformational changes of melibiose permease from Escherichia coli studied by infrared difference spectroscopy.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.
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description
onderzoeker
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researcher ORCID: 0000-0002-8393-2721
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name
Xavier León
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Xavier León
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Xavier León
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Xavier León
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type
label
Xavier León
@ast
Xavier León
@en
Xavier León
@nl
Xavier León
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prefLabel
Xavier León
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Xavier León
@en
Xavier León
@nl
Xavier León
@sl
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P21
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0000-0002-8393-2721