Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice
H Beard, AJ Luck, S Hassiotis, B King, PJ Trim… - Molecular genetics and …, 2015 - Elsevier
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, neuroinflammation and degeneration. Pre-clinical animal studies
indicate that replacement of the deficient enzyme, sulfamidase, via intra-cerebrospinal fluid
(CSF) injection is a clinically-relevant treatment approach, reducing neuropathological …
cognitive impairment, sleep–wake cycle disturbance, speech difficulties, eventual mental
regression and early death. Neuropathological changes include accumulation of heparan
sulfate and glycolipids, neuroinflammation and degeneration. Pre-clinical animal studies
indicate that replacement of the deficient enzyme, sulfamidase, via intra-cerebrospinal fluid
(CSF) injection is a clinically-relevant treatment approach, reducing neuropathological …