Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice

MPS IIIA is an inherited neurodegenerative lysosomal storage disorder characterized by cognitive impairment, sleep–wake cycle disturbance, speech difficulties, eventual mental regression and early death. Neuropathological changes include accumulation of heparan sulfate and glycolipids, neuroinflamma...

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Veröffentlicht in:	Molecular genetics and metabolism 2015-05, Vol.115 (1), p.33-40
Hauptverfasser:	Beard, Helen, Luck, Amanda J., Hassiotis, Sofia, King, Barbara, Trim, Paul J., Snel, Marten F., Hopwood, John J., Hemsley, Kim M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Brain - pathology Cerebrospinal fluid Cisterna Magna Disease Models, Animal Drug Administration Routes Enzyme replacement therapy Enzyme Replacement Therapy - methods Heparitin Sulfate - cerebrospinal fluid Humans Hydrolases - administration & dosage Hydrolases - cerebrospinal fluid Infusions, Intraventricular Infusions, Spinal Injections Injections, Intraventricular Lysosomal storage disorder Mice Mice, Congenic Mice, Inbred C57BL Mouse MPS IIIA Mucopolysaccharidosis III - drug therapy Mucopolysaccharidosis III - genetics Recombinant Proteins - administration & dosage Recombinant Proteins - cerebrospinal fluid Sanfilippo syndrome
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