DELIVERING GENES TO THE BRAIN ENDOTHELIUM TO TREAT LYSOSOMAL STORAGE DISORDER-DERIVED NEUROPATHOLOGY

Applicants sought to express human iduronate-2-sulfatase (hIDS) in the brain endothelium of a mouse model of Mucopolysaccharidosis type II (MPSII, Hunter's syndrome) to enable enzyme secretion into the brain parenchyma. In this disorder, IDS deficiency results in the pathophysiological accumula...

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Hauptverfasser:	BOTTICELLO-ROMERO, Nuria R, WU, Jason, BEDDOW, Thomas, DEVERMAN, Benjamin
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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