Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis. [electronic resource]
Producer: 20130129Description: 213-21 p. digitalISSN:- 1096-7206
- Animals
- Brain -- metabolism
- Disease Models, Animal
- Enzyme Replacement Therapy
- Female
- Humans
- Lysosomes -- drug effects
- Male
- Mice
- Mice, Knockout
- Motor Activity -- drug effects
- Neuronal Ceroid-Lipofuscinoses -- drug therapy
- Recombinant Proteins -- administration & dosage
- Rotarod Performance Test
- Thiolester Hydrolases -- administration & dosage
- Viscera -- drug effects
No physical items for this record
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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