Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder

Experimental Neurology

Volumn 278, Issue , 2016, Pages 11-21

  King, Barbara ^a   Setford, Meghan L ^a   Hassiotis, Sofia ^a   Trim, Paul J ^a   Duplock, Stephen ^a   Tucker, Justin N ^a   Hattersley, Kathryn ^a   Snel, Marten F ^a   Hopwood, John J ^a   Hemsley, Kim M ^a  

^a SOUTH AUSTRALIAN HEALTH AND MEDICAL RESEARCH INSTITUTE   (Australia)

Author keywords

Cerebrospinal fluid; Enzyme replacement therapy; Lysosomal storage disorder; Mouse; MPS IIIA; Osmotic pump

Indexed keywords

GANGLIOSIDE GM3; HEPARAN SULFATE SULFATASE; HYDROLASE; N-SULFOGLUCOSAMINE SULFOHYDROLASE; SULFATASE;

ANIMAL; BRAIN; C57BL MOUSE; COMPLICATION; DISEASE MODEL; DOSE RESPONSE; DRUG ADMINISTRATION ROUTE; DRUG DELIVERY SYSTEM; DRUG EFFECTS; FEMALE; GENETICS; GM2 GANGLIOSIDOSIS; HUMAN; MALE; MAZE TEST; METABOLISM; MOTOR ACTIVITY; MOUSE; NEURODEGENERATIVE DISEASES; PATHOLOGY; SANFILIPPO SYNDROME; TRANSGENIC MOUSE;

ANIMALS; BRAIN; DISEASE MODELS, ANIMAL; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION ROUTES; DRUG DELIVERY SYSTEMS; FEMALE; G(M3) GANGLIOSIDE; GANGLIOSIDOSES, GM2; HUMANS; HYDROLASES; MALE; MAZE LEARNING; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MOTOR ACTIVITY; MUCOPOLYSACCHARIDOSIS III; NEURODEGENERATIVE DISEASES; SULFATASES;

EID: 84956603358     PISSN: 00144886     EISSN: 10902430     Source Type: Journal    
DOI: 10.1016/j.expneurol.2015.11.013     Document Type: Article

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